News

Global Genes Welcomes Applicants for RARE Meet-Ups Impact Grants

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…

Prepare to Light Up Buildings for Rare Disease Day 2022

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…

LEMS Case Tied to Rare Subtype of Lung Cancer 

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 Will Seek FDA Approval of Firdapse for Children

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…

Rare Disease Groups in US Join in Plea for Care Across State Lines

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…