News

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

Firdapse (amifampridine) improved muscle strength and response to nerve cell stimulation in adults with Lambert-Eaton myasthenic syndrome (LEMS), a pooled analysis of six clinical trials demonstrated. The findings showed that all six previous trials together “showed significant efficacy, consistent with earlier reports of beneficial efficacy” of Firdapse —…

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.

A federal appeals court has ruled that the U.S. Food and Drug Administration (FDA) should not have approved Ruzurgi (amifampridine) for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS). The decision by the U.S. 11th Circuit Court of Appeals is a win for Catalyst Pharmaceuticals, which…

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…

Cholinesterase inhibitors may be useful in controlling the symptoms of Lambert-Eaton myasthenic syndrome (LEMS) in people with relatively mild disease, as highlighted in a recent case report. The findings suggest that these medications may be particularly useful for managing LEMS in countries where Firdapse (amifampridine) — the…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Jacobus Pharmaceutical has issued a voluntary recall for three lots of Ruzurgi (amifampridine), a medication that is approved in the U.S. to treat people with Lambert-Eaton myasthenic syndrome (LEMS) ages 6–17. According to the company’s announcement, while doing confirmatory full testing on one of the lots, control…