Denali and Sanofi Partner to Advance Small Molecules That Might Treat ALS, Other Diseases

Denali Therapeutics and Sanofi have joined efforts to develop small molecule inhibitors of the RIPK1 enzyme, a new class of potential therapeutic agents for a range of neurological and inflammatory diseases, including amyotrophic lateral sclerosis (ALS). The collaboration agreement is focused on the clinical development of two…

People with multiple rare mutations in genes known to cause amyotrophic lateral sclerosis (ALS) are significantly more likely to develop this disease at younger ages, a study in Japanese patients reports. The study, “Burden of rare variants in causative genes for amyotrophic lateral sclerosis (ALS) accelerates…

While TDP-43 aggregates are associated with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, researchers have found that these protein clumps are also involved in the regeneration of  healthy skeletal muscle. This finding — that abnormal TDP-43 aggregates temporarily form to repair damaged muscle — may open new lines of research into…

Extracts of velvet bean and Indian ginseng, two plants commonly used in the Ayurvedic medicine of India, were found to ease sleep disturbances and hyperactivity in a fruit fly model of amyotrophic lateral sclerosis (ALS), while also rescuing locomotor function. The study, “Standardized phytotherapic extracts rescue anomalous locomotion and electrophysiological responses…

Stem cell therapy seems to be safe in patients with amyotrophic lateral sclerosis (ALS) and clinical trials evaluating its effectiveness are on the way, according to a review study. The review, “Stem cell transplantation for amyotrophic lateral sclerosis,” was published in the journal Current Opinion in…

Have you heard the news? They’re banning plastic straws. Oh, darn! Just when I thought I had my ALS symptoms all figured out, perfect solutions in place, and solid daily routines to follow, a new curveball comes my way. The fact is I need straws! Not just any…

Integrated care is a definite advantage of multidisciplinary clinics according to amyotrophic lateral sclerosis (ALS) patients, but many complain about travel and mobility impediments as major barriers to attend such clinics, a Canadian study reports. Many patients also look at multidisciplinary clinics as potential ALS patient advocates to inform health…

Treatment with a compound called inosine over 12 weeks increased the levels of the antioxidant urate, a neuroprotective agent, in patients with amyotrophic lateral sclerosis (ALS), according to a pilot trial. The investigational therapy also showed a positive safety and tolerability profile, indicating it might have potential as an…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…

Researchers have discovered that C9orf72 — the gene whose mutated version is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) — works by activating key proteins for the transport and degradation of molecules inside the cell, in normal conditions. This discovery expands knowledge on how C9orf72 may contribute to the…