News

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…

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…

Immunofluorescence, a technique that can be used to detect antibodies associated with Lambert-Eaton myasthenic syndrome (LEMS), was negative in a 72-year-old woman, but another technique — called immunoblot — was positive for LEMS-associated antibodies. This ultimately allowed for a LEMS diagnosis and the discovery of a previously unknown tumor.

A woman with Lambert-Eaton myasthenic syndrome (LEMS) twice tested positive for a rare but high-risk blood marker of cancer, called type 2 antineuronal nuclear antibody (ANNA-2), while no tumor was evident, according to a case report. While LEMS often occurs in the context of cancer, repeat screenings over…

Catalyst Pharmaceuticals has secured a second U.S. patent covering Firdapse (amifampridine), an approved oral treatment for Lambert-Eaton myasthenic syndrome (LEMS). “We are pleased that this second patent for Firdapse has been issued and believe that it further enhances our intellectual property protection for Firdapse,” Patrick J. McEnany, chairman…

Health Canada has re-issued its approval of Ruzurgi (amifampridine) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 and older. The decision comes two months after the Federal Court in Canada cancelled the therapy’s approval in the country due to data protection-related issues filed by Catalyst Pharmaceuticals, which commercializes…

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…

Catalyst Pharmaceuticals has teamed up with DyDo Pharma to develop and commercialize Firdapse (amifampridine) in Japan for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). Under the terms of their agreement, DyDo will be responsible for funding all clinical, regulatory, marketing, and commercialization activities in Japan. In turn,…