#NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says

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 ALS Association was founded in 1985 and is dedicated to helping patients who suffer from amyotrophic lateral sclerosis (ALS). The non-profit organization operates with a global scope, coordinating their research with some of the world’s top scientists. Here are five more things this incredible organization does: Research…

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…

In this video from the Brattleboro Reformer, learn more about how ALS patients like Andy are diagnosed from a doctor’s point of view. MORE: Andy shares his ALS journey in this touching video Neurologist Dr. Elijah Stommel explains that he sees two newly diagnosed ALS patients each week and that each…

In this EmpowerRN video, Caroline, a registered nurse, talks through the signs, symptoms and treatments for amyotrophic lateral sclerosis (ALS) and how the disease progresses. MORE: Seven facts about amyotrophic lateral sclerosis you might find interesting  The video is primarily aimed at fellow nurses, offering tips on studying…

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,…

Quick! Guess where I was the other day? Hint: I spent quality time with a few ghosts, goblins and vampires. Nope, I didn’t visit a haunted house – I went for a haircut! You sure can’t tell from the outside, but where I go has an explosion…