Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

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…

  Being diagnosed with amyotrophic lateral sclerosis (ALS) can be devastating for patients and their families and friends, but there are ways to move forward. We’ve put together a list of coping strategies to help you get through the first few weeks with help from the Mayo Clinic and alswindsor.ca. Allow time and…

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…

Amyotrophic lateral sclerosis (ALS) is sometimes known as Lou Gehrig disease after the baseball player who famously suffered from it. Although ALS is a more common disease than we would like it to be, there is still little known about it and many are unaware of the details of the condition. MORE: Find out…

ALS is a disease that progressively shuts down your muscles by attacking certain cells in the brain and spinal cord needed to keep our muscles moving. Eventually, patients will lose their mobility, have trouble moving, walking, swallowing and even breathing. There is no known cure for ALS and the treatments available…

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…