News

BREN-02, a lab-made form of the human protein engrailed 1 (EN1), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) as a potential treatment of amyotrophic lateral sclerosis…

A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…

The ALS Association and I AM ALS have filed a petition with the U.S. Food and Drug Administration (FDA), calling on the regulatory agency and Amylyx Pharmaceuticals to make AMX0035 available to people with amyotrophic lateral sclerosis (ALS) as quickly as possible. Signed by more than 50,000 patients, their caregivers and family, the…

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…

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…