The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. The 30-minute session can be accessed on the MDA’s Facebook page, and starts at 6 p.m. EST. People with…
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…
What an emotional roller-coaster time we’re in right now! During this COVID-19 pandemic, I’ve felt the upswings of coping and adapting well, followed by the downswings of worry that this crisis would never end. Only when I took a step back, to reflect on my feelings and identify what…
Radicava (edaravone), an approved treatment for amyotrophic lateral sclerosis (ALS), can be taken for up to one year without losing its effectiveness, and benefits patients who delay its start — say, after six months of placebo in a clinical trial, a post hoc analysis of that Phase 3…
Social distancing? Shelter in place? For many like me who live with ALS, the recent COVID-19 guidelines to “stay home” and “work from home” are what we already do! Every. Single. Day. Getting out and about for a quick trip to the grocery store or a meal at…
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…
A molecule that prevents cell death was able to prevent and reverse clumping of a mutant form of the protein superoxide dismutase 1 (SOD1) — the underlying cause of some familial amyotrophic lateral sclerosis (ALS) cases. …
Muscle cell activity helps to promote motor nerve cell repair after injury, scientists found in using a device to model motor neuron and muscle cell interaction. They believe this finding could be relevant to diseases like amyotrophic lateral sclerosis (ALS) that imperil motor neuron survival. The device was described…
Amyotrophic lateral sclerosis (ALS) patients with mild to moderate disease progression show an ability to drive that’s comparable to other adults, despite weaknesses with movement and cognition, a small study using a driving simulator suggests. The study included only patients who were still driving, and its researchers caution that…
Studies in animal models investigating mitochondrial abnormalities in amyotrophic lateral sclerosis (ALS) found that therapies targeting mitochondria — the powerhouses of cells — are consistently effective in prolonging survival, a review concluded. The review study, “Targeting…
