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) —…
News
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…
Eight patients will receive jacifusen, an experimental therapy for amyotrophic lateral sclerosis (ALS) caused by FUS gene mutations, under a joint effort from the ALS Association, Project ALS, and Columbia University’s Eleanor and Lou Gehrig ALS Center. The new clinical research program, which will be supported…
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…
Like many couples, Joe and Linda Lacroix of Milton, Vermont, spent much of their lives working, raising families, maintaining their home, and planning for the future. Unlike most, however, there was always a dark cloud looming on their horizon — a gene for Huntington’s disease carried by Joe’s mother.
Skin cells from people with amyotrophic lateral sclerosis (ALS) have altered metabolisms and increased levels of a type of cellular stress called oxidative stress, a new study shows. This may indicate new treatment targets for ALS. The study, “ALS skin fibroblasts reveal oxidative stress and ERK1/2-mediated…
A machine learning algorithm has shown that depression and a perceived lower quality of life are significant predictors of high caregiver burden among those who care for patients with amyotrophic lateral sclerosis (ALS), a study has found. The study, “Prediction of caregiver burden in amyotrophic lateral sclerosis:…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
Recent Posts
- Rollator revamp needed because design matters for living well with ALS
- Study probes high ALS risk in elite athletes and other ‘champions’
- First ALS patient dosed in study of experimental gene therapy VTx-002
- New collaboration targets protein buildup linked to brain diseases
- New funding backs commercial readiness efforts for ALS therapy