News

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,…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Almost all people with Lambert-Eaton myasthenic syndrome (LEMS) have generalized symptoms at disease onset, according to an analysis of data from a Dutch patient registry. That finding, and others, were similar between patient-completed questionnaires and medical records, which supported…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Plasmapheresis, or plasma exchange, may be an effective way of managing small cell lung cancer-associated Lambert-Eaton myasthenic syndrome (LEMS) and paraneoplastic cerebellar degeneration (PCD), combined with peripheral nerve damage, a case report suggests. The simultaneous development of LEMS and PCD is usually associated with small cell lung cancer (SCLC)…

RARE-X, a collaborative platform for patient-controlled data collection, global data sharing and analysis, is partnering with the Broad Institute of MIT and Harvard to advance and support the discovery of new treatments for 9,500 rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS). The collaboration will leverage Broad Institute’s…