News

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…

Immunotherapy Did Not Worsen LEMS in Patient With Lung Cancer

A patient with extensive-stage small cell lung cancer and Lambert-Eaton myasthenic syndrome (LEMS) saw a marked decrease in his lung tumors following treatment with a combination of an immune checkpoint inhibitor and chemotherapy — all while having no LEMS flare-ups. That finding from a recent case report, according to…

RARE-X, Global Genes to Help Collect Rare Disease Patient Data

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…