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More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

Columbia University medical researchers have received a grant from the Chan Zuckerberg Initiative, named after Facebook founder Mark Zuckerberg and his wife Priscilla Chan, to construct an atlas of gene activity in all cells in the spinal cord. The atlas has the potential to become a reference for investigators researching injuries…

Voyager Therapeutics has recently presented data of its leading adeno-associated virus (AAV) gene therapy for several neurological diseases, including amyotrophic lateral sclerosis (ALS), at the Congress of the European Society of Gene and Cell Therapy (ESGCT), Oct. 17-20 in Berlin, Germany. In their poster, “Translation of Intrathecal Delivery of an AAV Gene…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

The presence of diabetes is associated with a 70 percent reduction in the risk of amyotrophic lateral sclerosis (ALS), new study shows. The study, titled “The role of pre-morbid diabetes on developing Amyotrophic Lateral Sclerosis,” was published in the European Journal of Neurology. ALS patients tend…

BrainStorm Cell Therapeutics announced that the first patients have been enrolled in the Phase 3 clinical trial of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). NurOwn is a mesenchymal stem cell (MSC) platform created for the treatment of ALS. Because it is made from MSCs,…

Ipsen Biopharmaceuticals, an affiliate of Ipsen, announced that five abstracts discussing Dysport (abobotulinumtoxinA) were presented at the American Academy of Physical Medicine and Rehabilitation (AAPM&R) Annual Assembly, held Oct. 12-15 in Denver, Colorado. Dysport, a botulinum toxin product, is approved for treatment of spasticity, or shaking, in adult patients, which…

Changes in the numbers of immune cells in the blood are associated with the progression of ALS, a study reports. Keeping tabs on the numbers could help doctors track the disease and researchers identify targets to develop treatments around, it said. The study, “Correlation of Peripheral Immunity…

Participating in the 2017 Annual Northeast ALS Consortium (NEALS) meeting in Florida recently, biotech company Oxford BioDynamics presented two different epigenetic signatures for the diagnosis and prognosis of amyotrophic lateral sclerosis (ALS). The presentation was a result of a prospective study to determine the sensitivity and specificity of two…

Although there’s no cure for amyotrophic lateral sclerosis, managing it wisely can increase survival and improve patients’ and their families’ quality of life. One tool for managing it is a feeding tube. An article in the journal Neurology covered the pluses and minuses of using the tubes in ALS. It…