PrimeC Earns Orphan Drug Designation in Europe

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…

Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health and functioning of their motor neurons, according to the interim trial data. These data are still blinded,…

“I forgot how dark it is all the time,” my brother commented when he visited us in Michigan’s Keweenaw Peninsula over the holidays. The sun was trapped behind cloud cover for the entire week he was here, and some years, it only peeks out occasionally during January with near daily…

Spinogenix has been awarded a research grant from the U.S. Department of Defense (DOD) to further test its lead compound as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced. “We are pleased that the DoD has recognized the potential of our novel drug candidate to…

“There’s nothing but twigs and seeds, twigs and seeds, And they sure don’t deliver the punch that this ole head needs.” Jesse Winchester is lamenting his discovery that all that remains in his “medicinal” stash are the…

A system using human cells to model how the brain controls muscle movement has been developed for the first time, and scientists expect it to aid in understanding neurological disorders that affect movement, including amyotrophic lateral sclerosis (ALS). The study describing their model system, “Generation of Functional…

Mitsubishi Tanabe Pharma America (MTPA) has launched an extension study to assess the long-term effects of an investigational oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS). “As a company, we strive to always put patients first in our efforts to understand this debilitating…