News

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Catalyst Pharmaceuticals is planning to request that the approved use of Firdapse in the U.S. be expanded to include children with Lambert-Eaton myasthenic syndrome (LEMS). The company also announced, as part of its “patient first” commitment, an intention to sustain established access programs so that anyone with LEMS seeking…

A federal appeals court in the U.S. has issued a mandate that effectively vacates the 2019 regulatory approval of Jacobus Pharmaceutical’s Ruzurgi for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS). The ruling, by the U.S. Court of Appeals for the 11th Circuit, directs a lower district court…

The National Organization for Rare Disorders (NORD) has expanded its hands-on tools and courses for organizations focused on rare diseases, such as Lambert-Eaton myasthenic syndrome (LEMS). Supported by the Chan Zuckerberg Initiative, NORD’s RareLaunch program provides patients and caregivers with the tools necessary to launch a nonprofit. It also…

Investment in companies developing treatments for Lambert-Eaton myasthenic syndrome (LEMS) and other rare diseases increased by 28% in 2021 compared with the previous year, according to a new report from the rare disease nonprofit Global Genes. “Rare diseases continue to have a strong allure to investors, as evidenced…

A federal appeals court has denied Jacobus Pharmaceutical’s petition to rehear its prior ruling overturning the U.S. approval of the company’s Ruzurgi to treat children with Lambert-Eaton myasthenic syndrome (LEMS). The decision is a victory for Catalyst Pharmaceuticals, which sued the U.S. Food and Drug Administration over Ruzurgi’s approval…

Global Genes is accepting applications for its 2022 RARE Meet-Ups Impact Grant program, which provides funding for in-person meetings specifically tailored to better educate and assist patients, caregivers, and advocates for rare diseases like Lambert-Eaton myasthenic syndrome (LEMS). Funded meet-ups need to take place between April and November, the…

Seven new U.S. states have launched a Rare Disease Advisory Council, or RDAC, in the last year with the support of the National Organization for Rare Disorders (NORD), an advocacy group for the millions of Americans living with rare diseases, to include Lambert-Eaton myasthenic syndrome (LEMS). It is…

Since late November and embracing the holiday spirit, the National Organization for Rare Disorders (NORD) has been sharing a series of previously exclusive videos from its 2021 Living Rare Living Stronger Patient and Family Forum, aiming to improve the lives of people with rare diseases and their families.

Catalyst Pharmaceuticals has announced its partner DyDo Pharma has launched a Phase 3 registrational trial in Japan to evaluate the safety and effectiveness of Firdapse (amifampridine) to treat Lambert-Eaton myasthenic syndrome (LEMS). A registrational trial is designed to obtain sufficient clinical data to support the filing…