Forest Ray PhD,  —

A new project run by a consortium of European institutions aims to improve the care of people with amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS) through targeted use of artificial intelligence (AI). Called BRAINTEASER, the four-year study will monitor some 300 participants using various wearable sensors and…

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…

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…

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…

A pivotal clinical trial evaluating pridopidine for the treatment of amyotrophic lateral sclerosis (ALS) has enrolled its first participant, Prilenia Therapeutics, the medicine’s maker, announced. The Phase 2/3 study, dubbed HEALEY ALS Platform – Regimen D Pridopidine (NCT04615923), will compare the safety and effectiveness of…

Cytokinetics is preparing a global Phase 3 trial that will enroll more than 500 people with early amyotrophic lateral sclerosis (ALS) to test its investigational therapy reldesemtiv in slowing progressive muscle weakness, the company announced. Details from the trial design were shared at the 31st International Symposium…

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Seelos Therapeutics‘ SLS-005 (trehalose), a medication designed to slow the progression of amyotrophic lateral sclerosis (ALS), the company announced in a recent press release. The designation…

A review of Radicava (edaravone), an approved amyotrophic lateral sclerosis (ALS) treatment, found the medication generally well tolerated in real-life use by patients in six countries, matching reports from clinical trials. But its effectiveness at delaying disease progression was less evident, with findings of greatest efficacy coming from…

The Phase 3 clinical trial investigating an oral suspension formulation of edaravone (MT-1186) in treating amyotrophic lateral sclerosis (ALS) is now fully enrolled, the therapy’s developer, Mitsubishi Tanabe Pharma America, announced. This open-label study (NCT04165824) first opened in…