About a week after my initial ALS diagnosis, the crippling fog that it had produced lifted enough for me to seek a second opinion. The appointment took place at the neurology department at the University of North Carolina…
Adding MDA’s Vast Resources to My ALS Arsenal and Giving Back
Collaborative Medicinal Development (CMD) has enrolled the first patient in its Phase 2/3 clinical trial assessing the safety and efficacy of CuATSM, an investigational therapy for amyotrophic lateral sclerosis (ALS). The trial (NCT04082832) is aimed at evaluating the efficacy and safety of CuATSM. The…
Mexiletine, an oral medicine to treat irregular heartbeats, is safe and effective for reducing muscle cramps in patients with amyotrophic lateral sclerosis (ALS), results from a Phase 4 clinical trial show. To date, this is the only treatment shown to reduce both the number and intensity of cramps…
BrainStorm Cell Therapeutics announced that the Phase 3 clinical trial of its cell therapy candidate NurOwn in 200 amyotrophic lateral sclerosis (ALS) patients is fully enrolled and treatment is underway. The randomized, double-blind and multicenter trial (NCT03280056) is assessing the safety and effectiveness of three administrations of NurOwn into…
A Harvard Medical School professor and scientist, Sabrina Paganoni, has won the 2019 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Scientific Impact Award for her work in a Phase 2a trial testing fingolimod as a potential treatment for amyotrophic lateral sclerosis (ALS). Fingolimod is marketed as Gilenya (by Novartis), an…
Before my husband, Todd, got ALS, we had minor problems, but we were full of hope. We had a small baby so I wasn’t getting much sleep, but I knew that things would get better. Todd was sometimes frustrated with his work, but he hoped to be promoted. Our obstacles…
The experimental treatment SBT-272 delayed the onset of neurological symptoms, lowered levels of a biomarker of nerve damage, and prolonged the life of male mice in a model of amyotrophic lateral sclerosis (ALS). Stealth BioTherapeutics, the company developing SBT-272, also announced plans to open a Phase 1…
From time to time, a comment in response to one of my columns reveals that the commenter has lost someone to ALS. The first time it happened, I paid it little mind. Subsequent occurrences aroused my curiosity as to why a survivor would continue to thoughtfully remain current…
The final amyotrophic lateral sclerosis (ALS) patient treated with oral AMX0035 in the CENTAUR study has completed all scheduled follow-up visits and researchers will now evaluate data collected on the potential treatment’s safety and efficacy, Amylyx Pharmaceuticals, its developer, announced. Final results of the Phase 2 study (NCT03127514), which…
In last week’s column, I wrote about a gala banquet that I recently attended. As the event approached, I worried about what I would wear and how others would react to my mobility scooter. But my greatest concern was that I was one of the evening’s honorees. The…
Recent Posts
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- Early-career ALS scientist wins first MDA Research Momentum Award