News

People with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MNDs), as well as their caregivers and healthcare professionals, are being recruited across the U.K. to provide input for the development of a web-based tool to help patients decide if they want a feeding tube. Patients are being…

Boston Children’s Hospital (BCH) is renaming its amyotrophic lateral sclerosis (ALS) Augmentative Communication Program to the Jay S. Fishman ALS-Augmentative Communication Program at Boston Children’s, the hospital announced. Fishman was CEO of the American insurance firm Travelers Companies. A vocal…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

Indiana motorists can now sport a specialty license plate on their vehicles to show their support for amyotrophic lateral sclerosis (ALS), while contributing to the work into this neurodegenerative disease underway at the ALS Therapy Development Institute (ALS TDI). Proceeds from plate purchases will go to support studies taking place at…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Mitsubishi Tanabe Pharma America announced changes to enhance its out-of-pocket assistance program for people with amyotrophic lateral sclerosis (ALS) using Radicava (edaravone). The changes, aimed at making the program easier and more accessible, include a new “hybrid” card meant to help patients obtain appropriate pharmacy and medical co-pay…

To mark what would have been the 79th birthday of the theoretical physicist Stephen Hawking, who lived with amyotrophic lateral sclerosis (ALS) for 55 years, Answer ALS is providing researchers with free access to the world’s largest ALS data portal. To help spread the word, the organization…

Mexiletine, a heart medication used off-label to treat muscle cramps due to amyotrophic lateral sclerosis (ALS), was found to be well-tolerated and to stabilize the “hyperexcitability” of motor neurons in a small group of ALS patients in a pilot Phase 2 trial. Data from the study (NCT02781454)…

Imaging data of brain inflammation in people with amyotrophic lateral sclerosis (ALS) that is obtained using different tracers and imaging equipment can be combined using a technique called the “pseudo reference region approach.” This finding may make it easier to conduct multi-center studies about ALS, according to a recent…

The European Medicines Agency has granted orphan drug designation to PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS). PrimeC, developed by NeuroSense Therapeutics, also has received orphan drug designation in the United States. The designation, both in the U.S. and Europe, encourages drugmakers to develop…