News

Dimethyl fumarate or DMF — an oral therapy approved for multiple sclerosis (MS) — was well-tolerated among adults with amyotrophic lateral sclerosis (ALS) but failed to significantly slow disease progression or to improve survival and quality of life in these patients, according to data from a Phase 2…

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

Expansion Therapeutics has raised $80 million in funding to support the development of its small molecule RNA platform, called SMiRNA, for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The Series B financing will help the drug development company use its platform to discover small molecules targeting RNA…

Research into gene therapies for motor neuron diseases (MNDs), a group of progressive neurological disorders that includes amyotrophic lateral sclerosis, is set to begin at the University of Sheffield in collaboration with Cell and Gene Therapy Catapult. Supported by a £513,141 (about $700,00) grant from LifeArc and the…

Cytokinetics is accepting applications from advocacy groups working in cardiovascular and neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), for grants worth $20,000 each that will help them expand their communications and community engagement. In total, five Cytokinetics Communications Fellowship Grants will be awarded winning organizations. The deadline for applications…

The ALS Association has launched a new diagnostic guide — called thinkALS — to aid neurologists in more quickly suspecting and diagnosing amyotrophic lateral sclerosis (ALS) at its early stages. Early diagnosis will allow patients to join clinical trials, and to access treatments and care at disease stages where there’s…

Bionews Insights is launching a survey, in collaboration with ALS News Today, with the goal of understanding how different aspects of this disease affect an individual’s quality of life. The survey, which is expected to take about 15 minutes to complete, opens with WHOQOL-100, a set of questions developed by…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have launched an expanded access program (EAP) in the U.S. to allow certain people with amyotrophic lateral sclerosis (ALS) to gain access to its experimental oral therapy CNM-Au8. Called CNMAu8.EAP02, the program will focus on patients who are not eligible…

Seelos Therapeutics’ experimental therapy SLS-005 (trehalose) will serve as the fifth arm of the multi-regimen HEALEY trial for amyotrophic lateral sclerosis (ALS), after being cleared by the U.S. Food and Drug Administration and the Mass General Brigham Institutional Review Board. With this final regulatory authorization, Seelos may still…