News

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…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

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…

The rare case of a man with advanced small cell lung cancer (SCLC) who went on to develop Lambert-Eaton myasthenic syndrome (LEMS) after receiving treatment with Tecentriq (atezolizumab), an immune checkpoint inhibitor, was described in a recent study. Researchers say this case highlights the need for vigilance in…

The recent case of a man with Lambert-Eaton myasthenic syndrome (LEMS) linked to a rare type of lung tumor, shows the condition can arise in patients with types other than small cell lung cancer. The case report, “Pulmonary Large Cell Neuroendocrine Carcinoma Associated With Lambert-Eaton Syndrome,”…

Catalyst Pharmaceuticals plans to file an application with the U.S. Food and Drug Administration (FDA) requesting that the use of Firdapse (amifampridine), an approved oral therapy for adults with Lambert-Eaton myasthenic syndrome (LEMS), be expanded to children. The announcement came after the company received a favorable decision from…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…