A branch of the European Medicines Agency (EMA) has recommended that Prilenia’s investigational therapy pridopidine be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Medicines with the potential to become safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
Exservan, the first oral film formulation of riluzole for treating amyotrophic lateral sclerosis (ALS), has been launched in the United States, its developer, Mitsubishi Tanabe Pharma America (MTPA), has announced. Rapidly dissolved when placed on top of the tongue, Exservan — specifically developed to help meet the needs…
Note: This story was updated June 30, 2021, to clarify that Exservan is placed on top of the tongue, not under it. Specialty pharmacy services for Exservan (riluzole), a twice-daily film that dissolves when placed on the tongue and approved to treat amyotrophic lateral sclerosis…
A previously unknown form of amyotrophic lateral sclerosis (ALS) — one with onset during childhood — is caused by mutations that alter the production of certain lipids (fat molecules), scientists report. “We found that a genetic form of the disease can also threaten children. Our results show for the first…
Cedars-Sinai Medical Center, in California, has been awarded an $11.99 million grant to support a clinical trial that will test specifically engineered neural progenitor cells as a potential therapy for amyotrophic lateral sclerosis (ALS). The work, funded by the California Institute for Regenerative Medicine, or CIRM, will build…
In a response letter to The ALS Association, the U.S. Food and Drug Administration (FDA) has recognized the unmet therapeutic need of people with amyotrophic lateral sclerosis (ALS) and reaffirmed its commitment to the 2019 ALS clinical trial guidance. However, the agency did not provide any details about how it…
Largely due to going virtual this year, the annual HayesDavidson 5K (HD5K) charity run met its goal of raising £40,000 (nearly $56,000) for the Motor Neurone Disease (MND) Association. The event previously was held in Hyde Park in London. However, the switch to the digital format enabled more…
Mutations in the FUS gene, a common cause of amyotrophic lateral sclerosis (ALS), impair the regeneration and growth of motor neuron extensions toward muscle cells, according to a study using a new miniaturized human model of neuron-muscle interactions. These deficits, along with the reduced number of nerve-motor connections, were lessened by blocking…
Most unpaid caregivers in the U.K. have not had any breaks from their caregiving role during the COVID-19 pandemic, with nearly three-quarters reporting feeling exhausted, stressed, and anxious, according to a survey of more than 2,800 caregivers conducted by Carers UK. During the pandemic, carers lost a mean…
Get regular updates to your inbox.
