Rare genetic mutations that affect functionally related proteins act in a synergistic manner to determine the course of ALS, a new genetic screening study shows. It also points to several new mutations that might lead to this disease. “We provide evidence for a number of entirely novel genetic variants of…
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WASHINGTON — Visitors to the ALS Association’s Washington, D.C., headquarters can’t help but notice the silver baseball bat sitting in a glass display case at the entrance. An inscription says the Louisville Slugger was presented in 1989 to Rodney L. Houts, the nonprofit group’s first president and CEO, to mark…
Anthony Senerchia Jr., the 46-year-old man who served as the inspiration for the ALS Ice Bucket Challenge, died on Nov. 25. He lived with amyotrophic lateral sclerosis (ALS) for 14 years. Senerchia was diagnosed with ALS shorty after marrying his high school sweetheart, Jeanette Hane, in 2003. In…
Analyzing muscles via magnetic resonance imaging (MRI) can help detect small physiological changes that happen when motor neuron diseases like amyotrophic lateral sclerosis (ALS) get worse, concludes a study by British researchers. The study, “Imaging muscle as a potential biomarker of denervation in motor neuron disease” appeared…
A blood test can be used to diagnose ALS and other neurodegenerative diseases, a study indicates. The biomarker development company DiamiR came up with the test, which measures levels of small RNA molecules in blood. It is much less invasive than other ways of diagnosing the diseases, according to researchers…
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…
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FDA-Approved Schizophrenia Medicine Orap Appears to Benefit ALS Patients, Canadian Study Reports
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…
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