#Motor Neuron Disease Treatment

[stemcelltherapyinindia]

Unraveling Hope: Stem Cell Therapy for Motor Neuron Disease (MND)

Discover Hope Unraveled! 🌟 Explore Stem Cell Therapy for Motor Neuron Disease (MND). Breakthrough Solutions for Improved Mobility and Quality of Life Await.

[brahmhomeo]

Guillain Barre syndrome treatment

[creativeera]

Amyotrophic Lateral Sclerosis Treatment: Current Perspectives on Amyotrophic Sclerosis Treatment

What is Amyotrophic Lateral Sclerosis Treatment? Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. The motor neurons, which are responsible for controlling voluntary muscle movement, gradually die in people with ALS. When the motor neurons die, it becomes very difficult to control muscle movement and eventually the muscles weaken and waste away. ALS usually affects people between the ages of 40 to 70 years. About 5-10% of ALS cases are considered familial, meaning they run in families. Most ALS cases are sporadic with no known family history. Men are slightly more likely than women to develop ALS. It is estimated that about 20,000 Americans may have the disease at any given time. Symptoms of ALS typically include slurred speech, muscle weakness, twitching or cramping, difficulty swallowing or breathing, and impaired walking or use of arms and hands. Most people with ALS lose their ability to speak, eat, move, and breathe within 3 to 5 years of diagnosis. The disease progresses differently in each individual and life expectancy can vary widely, with some people living for up to 10 years or more after symptoms first appear. Unfortunately, at this time there is no cure for ALS. Current Treatment Options While there is currently no cure for Amyotrophic Lateral Sclerosis Treatment, some treatments can help manage symptoms and quality of life for people living with the disease. The most common drug treatments for ALS include riluzole, edaravone, and dextromethorphan/quinidine. Riluzole (Rilutek) was the first FDA-approved drug for ALS in 1995. It is believed to work by blocking glutamate, a neurotransmitter that appears to play a role in motor neuron death. Riluzole has been shown to modestly extend survival by 2 to 3 months on average. Side effects may include nausea, fatigue, and liver problems. Edaravone (Radicava) was approved in 2017 as the second drug shown to slow functional decline in ALS. Clinical trials found edaravone reduced motor function decline over 6 months compared to a placebo. It is believed to work as an antioxidant, protecting cells from free radical damage. Side effects are generally mild and may include bruising, headache, and edema. Dextromethorphan/quinidine (Nuedexta) received FDA approval in 2011 as an add-on treatment for pseudobulbar affect, a condition that causes involuntary and unpredictable laughing or crying episodes unrelated to a person's feelings or mood. While not a treatment specifically for ALS motor symptoms, it can help manage this distressing symptom that sometimes occurs with ALS. Common side effects include diarrhea, nausea, dizziness, and vomiting. In addition to medications, other treatments that may help manage ALS symptoms include physical and occupational therapy to maintain mobility and function for as long as possible. Speech therapy can help address swallowing and speaking difficulties. Respiratory therapy and mechanical ventilation support, such as non-invasive ventilation, may improve breathing problems and quality of life. Nutritional supplements or feeding tubes help address weight loss or difficulty swallowing. Braces, walkers, and wheelchairs provide mobility assistance as needed. Stem Cell and Amyotrophic Lateral Sclerosis Treatment Exciting new avenues of research are exploring the potential of stem cell and gene therapies for ALS. Several clinical trials are investigating various approaches with the goal of replacing lost motor neurons or protecting and preserving existing motor neurons. One promising area involves transplanting motor neuron progenitor cells, stem cells that can develop into motor neurons, directly into the spinal cord. Early phase trials have begun testing the safety and potential benefits of this approach. Another trial is looking at transplanting olfactory bulb stem cells, which naturally migrate to the brain, into the lumbar region of the spinal cord. Get more insights on Amyotrophic Lateral Sclerosis Treatment

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Priya Pandey is a dynamic and passionate editor with over three years of expertise in content editing and proofreading. Holding a bachelor's degree in biotechnology, Priya has a knack for making the content engaging. Her diverse portfolio includes editing documents across different industries, including food and beverages, information and technology, healthcare, chemical and materials, etc. Priya's meticulous attention to detail and commitment to excellence make her an invaluable asset in the world of content creation and refinement.

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[vaidyayogeshvanii]

Top Motor Neuron Disease Treatment Doctors in Surat

You need look no farther than Vaidya Yogesh Vani for the Top Motor Neuron Disease Treatment Doctors in Surat. Our knowledgeable staff is committed to offering patients with motor neuron disorders complete care. We provide individualized treatment programs made to fit the requirements of every patient, guaranteeing the greatest results. To control symptoms and enhance quality of life, our experts apply cutting edge methods and the most recent findings. When it comes to treating motor neuron illness in Surat, Vaidya Yogesh Vani is a reputable option because of his kind demeanor and commitment to the welfare of his patients. Our top priority is your well-being.

[healthcare021]

Revolutionary Motor Neuron Disease Treatment in India

Diseases favor no one. Some of the most famous personalities of our times, such as Dr. Stephen Hawking and Mao Zedong had been inflicted with the disease. MND is an uncommon but deadly condition that affects the brain and nerves. The term MND is commonly used for a group of neural diseases wherein the nerves start degenerating, and eventually die. This causes the muscles to become weaker and weaker, eventually leading to paralysis. Doctors call this condition atrophy. Motor Neuron Disease Treatment in India includes a combination of therapies. Moreover, there is abundant evidence demonstrating the effectiveness of such treatments.

[derinthescarletpescatarian]

hey! so i hope this isn’t too creepy/nosey, but im a medical student and i was reading your possible fibromyalgia post and have a couple ideas lol. full important disclaimer that im only partly into my studies and im currently in the hypochondriac phase and also your summary was amazing but a real doc would ask way more questions, so please consult with an actual doc and take everything i say with a grain of salt! but like your symptoms aren’t nothing so i would def encourage finding a doc that you trust to do a proper exam and run some tests. also im operating under the assumption that you’re under 50 lol, bc if you’re over 50ish that’s a whole diff list of possible diagnoses.

so the thirst thing you’re talking about is often called polydipsia and is commonly associated with diabetes insipidus. that’s not the normal diabetes you think about, but happens when your body can’t regulate fluids in your body properly. id think of this if you’re also peeing a lot lol. your doc would have to do some kidney tests for that, which wouldn’t be part of the blood panel you mentioned. i’m a little skeptical that it’s hypokalemia bc that would’ve showed up on your blood test results. it could be transient electrolyte imbalances when you exercise so have one of those electrolyte packets when you exercise lol, bc it never hurts to try the easy solutions first, but chronic low potassium should’ve shown up? tho eating sweet potatoes has never hurt.

other things it could be is a lower motor neuron problem bc you mentioned twitches and muscle weakness which is typical for those. i def can’t say more without tests, but look into/get your doc to look into myasthenia gravis or LEMS and see if either of those fit. i think it’s possible bc these often also start with face/upper body symptoms, but would need way more questions/tests to know. it’s unlikely but could also be a glycogen storage disease called McArdle disease bc you describe a second wind thing when you exercise along with exercise intolerance. that’s super rare tho so it’s unlikely unless someone in your family has it/has similar symptoms.

also look into autoimmune stuff like rheumatoid arthritis, lupus, and sjögrens disease. i have way less useful info on that bc we haven’t gotten to it in class yet lol, but sjögrens looks promising bc you often get dry mouth with it, and it often goes along with rheumatoid arthritis which could explain the joint stuff possibly.

it’s also totally possible this is fibromyalgia, but i would be cautious diagnosing it bc it often comes with fatigue and cognitive stuff which you didn’t mention. it’s also more of a pain thing, and doesn’t include your twitches/dry mouth. it’s def possible, and it was def something i thought of when i saw your symptoms, but personally i would want to rule out other stuff first bc fibromyalgia is pretty vague and often a diagnosis of exclusion when other things don’t fit.

sorry for overwhelming you!! i just saw your post and was like hmmm those symptoms sound like Something. again take my advice with a big grain of salt, but i do really think it’s worth asking your doc about it and getting tests done, bc even if there aren’t cures there are def treatments to help with a bunch of this stuff. it doesn’t sound urgent, but at least from your post your symptoms don’t sound like run of the mill aches and pains. hope you figure stuff out!!

The problem with 'muscles don't work right ouchy and I am also tired' is that it's a symptom for Absolutely Everything That Can Be Wrong With The Body. Is it cancer? Is it a terrible diet and sleep schedule? Who knows!

The doctor ran a diabetes test with the blood panel and it came up negative, but I don't know if that checks for weird kinds of diabetes. (Diabetes does not run in my family until we get very old.) That test was memorable because I have stupid fragile veins that freak out and collapse at the mere sight of a needle so I had to get stabbed nine times, they didn't manage to get the middle reading at all, and in the end they resorted to just stabbing my thumb with one of those diabetes home blood test thingies and manually squeezing my blood out into a tube drop by drop.

I looked up polydipsia and I don't think I have that. I think I just prefer my mouth to be wetter than my salival glands want it to be. 🤷‍♀️I think most of my problems are probably not related to any rare chronic disease, but just run-of-the-mill autism making it hard to look after myself or properly notice and process my physical condition and adapt accordingly. I don't eat enough fresh foods because it's hard to plan with the very short timeframe to prepare and eat them in. I'm uncoordinated and damage my body a lot through overwork or using muscles incorrectly because autism makes it hard to keep track of those things. My mouth feels dry and my skin feels itchy and my muscles feel sore because that's what being autistic feels like. My sleep schedule is garbage because my executive function is garbage and even once I do manage to get myself into the bed I can't just "go to sleep", I pass out when I'm ready to pass out.

I'm not saying it's impossible for anything else to be going on, but I think the known factor is the simplest explanation here. It's 2:30pm and I've been putting off breakfast for five hours. Every time I go into the kitchen I get distracted by housework instead. I am very hungry. This is not behaviour that is conducive to a well-functioning body.

[mindblowingscience]

Electrical deep brain stimulation (DBS) is a well-established method for treating disordered movement in Parkinson's disease. However, implanting electrodes in a person's brain is an invasive and imprecise way to stimulate nerve cells. Researchers report in Nano Letters on a new application for the technique, called magnetogenetics, that uses very small magnets to wirelessly trigger specific, gene-edited nerve cells in the brain. The treatment effectively relieved motor symptoms in mice without damaging surrounding brain tissue.

Continue Reading.

[scotianostra]

Scottish Rugby Player Doddie Weir was born on July 4th in Edinburgh.

Born George Wilson Weir, Doddie was educated at Daniel Stewart’s and Melville College and given the school’s heritage with the sport, and Doddie’s size, it was only natural he would take up the rugby.

After his education finished he played for Stewart’s Melville FP, the FP stands for former pupil, he then went on to play for Melrose in the Scottish Borders, and was part of the team that won six Scottish club championships.

He was brought uup on Cortleferry Farm, near the village of Stow in the Scottish Borders. horses were a big part of his early life and he competed at local gymkhanas along with his sister Kirsty. Later he went on to represent Scotland at national equestrian events. Once, at the Scottish Horse Trials, he competed against Princess Anne.

As his rugby career progressed he moved to England in 1995 to join the Newcastle Falcons, helping them win the Premiership in 1998.

Lock Weir was capped 61 times by Scotland, and was part of the victorious Lions tour to South Africa in 1997.

He moved back to Scotland to join the newly reformed Borders team in 2002 where he remained until his retirement from professional rugby. He finished his playing career together with Gary Armstrong at Borders Reivers in 2004.

In 2016 Doddie was diagnosed with Motor Neurone Disease (MND) in 2016 and made his diagnosis public the following summer. Although the average life expectancy is between one and three years, he has defied the odds to keep battling and his charity, the My Name'5 Doddie Foundation, has raised more than £4million for research into a cure. Doddie was also told that a year later he would not be able to walk, he defied what the doctors said still walked in up to the final months of his life a testament to the determination and fighting spirit of this remarkable man.

A big favourite with the Murrayfield crowd, the 6ft 6in lock was famously described by the late commentator Bill McLaren as as being “on the charge like a mad giraffe”.

Doddie spoke of his MND

“As a bloke you just think ‘I’m fine’, but with this that’s not quite the case. Basically it’s a muscle wasting disease and that’s how in the later life of MND it’s horrific because you need help everywhere.

"Basically your muscles in your legs disappear so you can’t walk; you can’t really eat, and then your muscles within your speech disappear so you can’t speak; you can’t swallow and can’t breath so it’s horrific what happens.

”….“But it’s such a debilitating condition and there’s nothing out there that can help any patient with MND.

He has signed a “Do not resuscitate” (DNR) IN 2020.

In February 2020 Doddie said his decision to refuse potentially lifesaving CPR came after a tough chat with his sons Hamish, Angus, and Ben, "I’ve had to talk to them about DNR. We’ve just signed a document for that at the moment, which isn’t easy.”

He added: “You just have to be honest and open and they took it really well.”

Weir set up a charity called My Name's Doddie Foundation to help fund treatments for motor neurone disease.

In 2018 The Doddie Weir Cup was inaugurated, a perputual Rugby Union trophy to be played between Scotland and Wales, the first match in Cardiff in November that year was won by Wales, Scotland are the current holders.

Neither the Welsh Rugby Union nor the Scottish Rugby Union originally intended to contribute any of the gate receipts from the inaugural match to the charity, but pressure from fans and in the media resulted in them eventually donating a six-figure sum

On the evening of 26 November 2022, his family announced that he had died of motor neuron disease, aged 52. A memorial service was held at Melrose Parish Church on 19 December, with audio from the service relayed to Melrose RFC's ground The Greenyards.

Clinical trials into motor neurone disease (MND) have received a £500,000 boost from the My Name's Doddie Foundation to mark what would have been his 54th birthday.

[pacifistcowboy]

hey you ready for the most bonkers idea for my sol dimension ocs that i’ve had so far

so i’m thinking sol-rouge will have an older sibling who’s unwell in some way (might be motor neurone disease or based on it ‘cause my dad has that so i’m rather familiar with the symptoms and treatment but i probably won’t go too intense) ((also dw about that massive personal life bomb i just dropped, i also made sure my dad was okay with me applying our experiences into a story)) and sol-rouge tries to take care of them and support them both by just breaking into places for shelter or resources and sometimes fighting people if he gets caught in the act or is desperate enough. if they’re with him at the time, sol-rouge’s sibling will pipe up and try to ask people if sol-rouge can help in exchange for goods so he doesn’t immediately have to jump to just stealing, which is what he usually does ‘cause he’s so awkward and uncomfortable around people that he’d prefer to commit a crime than talk to anyone.

sol-rouge may or may not run into mittens during this time, or maybe they only meet later.

at some point shelly “ivy” organik will come across them and due to sol-rouge’s skill, seeming lack of morals, and desperation, she asks him to help her with her evil plans and in exchange she’ll help his sibling. at first sol-rouge refuses as he doesn’t believe ivy can help them, but using her chlorokinesis she creates some sorta tree-plant-machine-suit thing which helps his sibling in many ways including breathing n having a way to move around, n says they can use the plant-tree-mech thing as long as sol-rouge helps her, so he agrees.

sol-rouge’s sibling is probably very wary and hesitant about helping ivy, but the device she made helps them so much that they can’t really imagine just giving it back and struggling like before, so they go along with the plans, albeit very unconfidently. i think the tree-suit will have some form of weaponry so they can join sol-rouge in doing ivy’s bidding.

sol-rouge n their sib will either meet mittens for the first time or clash WAY more with her during this. things will probably escalate that blaze n stuff has to get involved, ivy probably also recruits glacier (i’ve decided sol-shadow’s name is glacier) so sol-rouge n their sibling are also working with her. stuff goes down.

eventually sol-rouge’s sibling will hit a point where they just morally can’t help ivy, and they choose to help the heroes stop her. glacier will also go through the discovery of how her parents actually sucked n that she’s on the wrong side so she’ll also join the heroes, but sol-rouge doesn’t really care about being good or bad, he just wants his sibling to be safe, but his sib is adamant about saving the people, so he joins them.

stuff happens, they beat ivy, and she tries to take her tree-suit-mech thing back, but literally everyone stops her n protects sol-rouge’s sibling from her, so she gets pissed n gives up. so now sol-rouge’s sibling permanently has a funky plant suit that alleviates their symptoms and they can walk around in! i’d say they’re scared ivy will constantly try to take it back, and might one day succeed, also they just care a lot about the innocent people of the world, so they’re determined to kill ivy. sol-rouge still doesn’t really care about people but his sibling is now healthier so he’s chill, plus he’s so close with them that he’s probably gonna have some of their compassion rub off on him.

anyway, really long-winded way of saying i’ve combined maria and omega into one character and it’s sol-rouge’s sibling. sol-omega isn’t a robot but a fella in a suit made by ivy. it’s bizarre but i think it works.

[princessanneftw]

Princess Anne will be visiting Kingsholm next week to send a rugby ball on a 555 mile journey. The Princess Royal will hand over a ball to rugby legends from across the UK.

And the move will kick off a a 200-strong peloton of cyclists on a poignant £500,000 charity fundraiser in memory of Doddie Weir, the Scottish rugby legend who died in November last year. A team of rugby legends will aim to cycle 555 miles in 48 hours to deliver it to Murrayfield Stadium ahead of the Scotland v Wales Six Nations game Saturday February 11 .

Once there they will hand the ball back to Princess Anne who is patron of the Scottish rugby team. The group, which includes former Scotland captain Rob Wainwright and World Cup winning former England skipper Martin Johnson, will raise money for motor neurone disease.

Son in law MIke Tindall is not on the official list of cyclists but earlier this year he paid tribute to Doddie who died while Mike was in the I'm A Celebrity....Get Me Out Of Here! jungle. He also donned his budgie smugglers to take part in Doddie Aid, a virtual exercise challenge taking place for six weeks from January 1.

The late Doddie Weir launched a fundraising foundation after being diagnosed with motor neurone disease (MND) in 2017. The Scotland v Wales match is known as the Doddie Weir Cup and this will be first time since he died in November 2022 at the age of 52, the match has taken place.

After collecting the ball from Anne the cyclists will travel via the Principality Stadium in Cardiff. Wainwright said: “This year marks the fourth anniversary of the Doddie Weir Cup, and it’s particularly poignant as it’s the first since Doddie’s passing in November, but this only drives us forward to continue making progress and raising awareness of MND.

“We have to believe that a breakthrough in MND treatment is just round the corner and push ourselves and others to raise funds to realise that dream."

[lifealteringstemcells]

Stem Cell Therapy: Recent Advancement For Spinal Cord Injuries

A spinal cord injury (SCI) is damage caused to the nerves in the spinal cords that are responsible for sending and receiving signals from the brain. The traumatic lesion in the spinal cord can cause permanent or irreversible sensory and motor deficits. Stem cell therapy has shown massive potential in repairing damaged tissues and promoting recovery of neurological function. Medical research shows the effectiveness of stem cell therapy in treating various neurodegenerative disorders like ischemic stroke, intracerebral hemorrhage, and spinal cord injury.

Causes Of Spinal Cord Injury

The increasing number of cases of spinal cord injuries worldwide has made SCI a global health priority. Sports injuries and road accidents have been recognized as the two common causes of SCI. Although traumatic conditions comprise major triggering factors to induce spinal cord damage, non-traumatic events such as inflammation, spinal disc degeneration, cancer, substantial tissue loss, and infections can also be the reason for injury to the spinal cord and peripheral nervous system,

Symptoms of Spinal Cord Injury

The emergency symptoms of SCI may include loss of control in body movements due to partial or complete loss of sensory function. It is characterized by loss of motor control of the back, arms, legs, and other body parts, leading to weakness and incoordination. Patients experience extreme pain and pressure in the back, neck, and head.

Other prominent symptoms that doctors have identified as effects of spinal cord damage are -

Difficulty in walking and sitting

Difficulty in breathing

Sudden reflexes or spasms

Loss of control over bladder and bowel movements

Lowered sexual sensitivity and infertility

Trouble in balancing

Numbness or tingling sensation in the hands, fingers, and feet

The Science of Stem Cell Therapy For Spinal Cord Injury Treatment

Spinal cord injury is a severe condition that damages the nerve cells and tissues in the spinal cord, resulting in paralysis or nervous function impairment. Current treatment approaches include medication, rehabilitation therapy, physical therapy, or surgery. However, most of them provide temporary relief and poor outcomes in the long run. Stem cell interventions have emerged as a promising treatment for SCI with its exemplary potential to repair and regenerate injured neurons and tissues.

The distinct ability of stem cells to proliferate and form any functional cell type makes them ideal for treating SCI. The cells self-renew into nerve cells to replace and repair the diseased cells. After they are introduced into a patient’s body, they reach the injured site and interact with the surrounding cells to produce neurotrophic growth factors and alter the microenvironment of the affected region in the spinal cord.

A preclinical study has shown that mesenchymal stem cells release growth-promoting factors that accelerate the growth of axons at the injured area and improve myelination (formation of specialized membranes around axons). Most importantly, the therapeutic potential of stem cells prevents further neuronal degeneration in the spinal cord, lowering inflammation and improving motor and sensory function.

Start Your Stem Cell Therapy Journey In Mexico

There is no better place than Mexico for stem cell therapy because this North American country is home to highly qualified and experienced medical professionals with a proven track record of successful cell therapies. Besides, the healthcare system in Mexico is also advanced, and its treatment solutions are most accessible and affordable to people all over the world.

Life Altering Stem Cell Therapy Institute is a trusted name if you are ready to receive stem cell therapy in Mexico. Their state-of-the-art technologies, innovative treatments, and comfortable clinic setup have earned them the tag of stem cell therapy best hospital in Mexico. Their health advocates are just a call away! Connect with them to learn more about stem cell therapy for treating spinal cord injuries and other chronic conditions.

[brahmhomeo]

Remove kidney stone without surgery

Day by day kidney stones problem are increasing and people are turning towards surgery. Even after surgery some complications remains and stone formation takes place. there is a precise medicated treatment for kidney stones which cures stones and also prevent its formation. renal calculi/kidney stones are the solid mass made up of crystals originate in kidneys result into intense pain around kidney region or/and along the line of urinary tract.

Brahm Homeopathic healing and research centre strength

highly experience team of doctors

high quality medicine from helios homeopathy pharmacy -LONDON.

Guided treatment with 24 hours availability for our patients for helping them.

proper follow up management till he get cured.

diet plan and life style correction according to disease he is suffering.

[bpod-bpod]

Delaying Tactics

A fatal neurodegenerative disease, amyotrophic lateral sclerosis (ALS) causes the progressive loss of motor neurons, cells that send signals from the brain to muscles. Patients gradually lose the ability to move, and ultimately even breathe on their own, often succumbing to respiratory failure. There is no cure, and existing therapies to mitigate symptoms have only limited effects, but new methods of drug discovery offer some hope. Researchers recently reprogrammed induced pluripotent stem cells derived from ALS patients (pictured) to make motor neurons, then used these to identify a potential drug candidate, known as ropinirole hydrochloride. Early clinical trials with small numbers of patients suggest the drug is safe, and that its protective effect on cultured cells translates to real benefits: taking the drug for 48 weeks led to slower declines in motor function, delaying disease progression. More extensive trials will help determine if this promising drug can yield new treatments.

Written by Emmanuelle Briolat

Image from work by Satoru Morimoto and Shinichi Takahashi, and colleagues

Department of Physiology, Keio University School of Medicine, Tokyo, Japan

Image copyright held by the original authors

Research published in Cell Stem Cell, June 2023

You can also follow BPoD on Instagram, Twitter and Facebook

[vaidyayogeshvanii]

BEST MOTOR NEURON DISEASE TREATMENT DOCTORS IN SURAT

Dr. Yogesh is the top motor neuron disease treatment doctor in Surat. Who are distinguished by their unmatched skills and kind behavior. Dr. Yogesh is an expert in the complex management of motor neuron disorders, combining cutting-edge treatments with a deep awareness of his patients' needs. He has established himself as a top specialist in Surat due to his commitment to enhancing the quality of life with personalized and successful treatment programs. Put your trust in Dr. Yogesh for excellent health care and an inspiring journey towards better health and well-being.

Top Motor Neuron Disease Treatment Doctors in Surat

[eretzyisrael]

Tofersen, which is injected into the spine like an epidural, is giving new hope to two patients at Tel Aviv Sourasky Medical Center

By NATHAN JEFFAY��Today, 11:49 am  

ALS patient Narkis Moshe with her doctor Prof. Vivian Dror of Tel Aviv Sourasky Medical Center (courtesy of Tel Aviv Sourasky Medical Center)

Israeli patients have become some of the first in the world to begin receiving a new ALS drug intended to slow the progress of the degenerative disease.

Tofersen, which is injected into the spine like an epidural, is giving new hope to two patients at Tel Aviv Sourasky Medical Center. One of them, Narkis Moshe of Nahariya, lost a brother to ALS, and was then diagnosed with the disease six months ago. She said that she is less scared about her fate now that she has started with the drug.

“When I was diagnosed with the disease I was very afraid because I didn’t want what happened to my brother to happen to me,” said Moshe, 58. “But it seems that today we’re entering a new era of research and treatment.

“I am very excited about the new treatment and I am hopeful that it will give me a good quality of life and many more years with my family.”

Tofersen, developed by Massachusetts-based Biogen, isn’t suitable for all ALS patients, as the disease has different underlying causes; rather, it is designed to help patients whose illness is cause by a faulty SOD1 gene.

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The drug has received the green light for a “compassionate use” program from the Food and Drug Administration, which permits it for specific patients in advance of full regulatory approval. Israel tends to follow the US in drug regulation, and is therefore allowing the drug to be used at Sourasky.

Illustrative image: neurones inside the human brain (onimate via iStock by Getty Images)

Prof. Vivian Dror, the Sourasky doctor who is administering the treatment, said that with only a few dozen patients worldwide receiving the drug so far, she is pleased that Israel is becoming an early adopter.

“It’s a potentially game-changing treatment,” she told The Times of Israel. “The patients receiving it are still communicative, able to swallow and with some use of their limbs, and it’s hoped that the drug will slow their deterioration and enable them to retain these abilities for longer.

“What it does is significant compared to the limited treatments that exist today for this disease.”

There are different underlying causes promoting the onset of ALS in different patients. Tofersen is designed specifically for those who suffer as a result of overproduction of hSOD1. It’s a protein produced by everyone but when produced in excess appears to have toxic effects on the motor neurons in the disease. A defect in the SOD1 gene, which produces hSOD1, is the cause of the problem.

Tofersen works by inhibiting the production of hSOD1, attempting to offset the effect of the SOD1 defect.

Dror said that while only a small minority of ALS cases are caused by the SOD1 gene, she believes that the underlying technology behind Tofersen will be adapted for patients who suffer as a result of other genes.

“It is important to note that this is a technological breakthrough not only for patients with this specific SOD1 defect, but also for other patients, because with the same technology it is possible to develop and create specific drugs for defects in additional genes,” she said. “This gives us hope.”