Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
BrainStorm Cell Therapeutics’ cell-based therapy NurOwn failed to significantly slow disease progression in people with rapidly progressing amyotrophic lateral sclerosis (ALS), top-line data from a Phase 3 trial show. The therapy, however, did result in clinically meaningful responses in a pre-specified group of patients with early ALS…
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 Asia…
The Motor Neurone Disease (MND) Association and the children’s charity Barnardo’s have launched a counseling service for young people in the U.K. whose family is affected by amyotrophic lateral sclerosis (ALS) or another motor neurone disease (MND). The service is expected to open counseling help to these…
A lightweight, wearable sensor is being developed that can detect minute facial movements and translate them into messages, potentially helping people with amyotrophic lateral sclerosis (ALS) communicate more easily. Once produced in mass, each device is also expected to cost about $10. The sensor is described in the study…
Transplanting a combination of two types of modified bone marrow cells eased motor deficits and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), researchers reported. Their study, “Enhancing the therapeutic efficacy of bone marrow-derived mononuclear cells with growth factor-expressing mesenchymal stem cells for ALS…
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…
AC Immune and WuXi Biologics have announced a partnership to advance the development of an investigational antibody treatment that targets the protein TAR binding DNA protein 43 (TDP-43) for treating certain central nervous system (CNS) conditions, including amyotrophic lateral sclerosis (ALS). Having identified and developed the anti-TDP-43…
The campaign for a league-wide annual Lou Gehrig Day recently received a major boost as all 30 Major League Baseball (MLB) teams endorsed an effort to honor the late New York Yankees star first baseman and raise awareness of amyotrophic lateral sclerosis (ALS). Lou Gehrig Day has…
Use of over-the-counter pain relievers, including acetaminophen and nonsteroidal anti-inflammatory drugs (NSAIDs) like ibuprofen and naproxen, significantly reduced the likelihood of developing amyotrophic lateral sclerosis (ALS), likely by working against inflammatory processes, a small meta-analysis reported. Aspirin, however, was not seen to affect…
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