News

Expressing “profound disappointment” with tirasemtiv — their lead candidate for amyotrophic lateral sclerosis (ALS) — officials at Cytokinetics have ended development of the once-promising therapy. Robert I. Blum, president and chief executive officer of the San Francisco-based biotech, announced negative results for VITALITY-ALS (NCT02496767)…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

The schizophrenia therapy Orap (pimozide) may be able to prevent the progression of ALS, Canadian researchers report. The team confirmed in a clinical trial of 25 ALS patients the results it saw in studies with worms, fish and mice. An antipsychotic drug, Orap is used to treat other psychiatric conditions…

The Muscular Dystrophy Association (MDA) is financing 13 new research programs aimed at developing treatments for amyotrophic lateral sclerosis (ALS) and other life-threatening diseases. Toward that goal, the organization recently announced it would distribute $3.5 million in new development grants. For the summer round of grants, 244 applicants submitted…

Neurimmune and Ono Pharmaceutical have entered a strategic partnership to develop human-derived monoclonal antibodies that might become the basis of new treatments for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS). Under the terms of the agreement, Neurimmune will lead research to generate and validate these antibodies using…

Patients with neurodegenerative diseases have views that are framed by the context of their lives, and end-of-life care should be equally personalized, respectful and given in a timely way, researchers in a study report. The study, “The views of adults with neurodegenerative diseases on end-of-life care: a…

Boston-based Flex Pharma has announced positive topline results from its exploratory Phase 2 trial testing investigational therapy FLX-787 in Australian amyotrophic lateral sclerosis (ALS) patients with frequent muscle cramps. The data showed that FLX-787 significantly reduced the intensity of cramp-associated pain and stiffness compared to placebo. The positive results support the…

Findings from a Phase 3 study of tirasemtiv to improve muscle strength — particularly of respiratory muscles — in amyotrophic lateral sclerosis (ALS) patients will likely be announced for a first time on Dec. 8 at the 28th ALS/MND meeting in Boston, a vice president of Cytokinetics, its developer, said…

Scientists have long linked abnormal versions of the protein TDP-43 to neurodegenerative diseases such as ALS, but they haven’t had a full understanding of how it causes nerve cell deterioration. A Japanese study in mice indicates that elevated levels of the normal version of TDP-43 can play a role in…

The Muscular Dystrophy Association (MDA) has awarded an MDA Venture Philanthropy (MVP) grant worth $233,200 to Iron Horse Diagnostics to support the development of a biomarker test for amyotrophic lateral sclerosis in the United States. The prognostic test, which was released in Europe in June under a license agreement…