News

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

A new podcast, called LEMS Aware, is launching this month on international Rare Disease Day with the goal of connecting members of the Lambert-Eaton myasthenic syndrome (LEMS) community and increasing awareness about the disease. The podcast will focus on topics unique to LEMS, but also of interest to the…

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.

People affected by Lambert-Eaton myasthenic syndrome (LEMS) and other rare diseases in the U.S. are encouraged to share their experiences through a new initiative called “What’s Your Story?” Submissions from patients, as well as caregivers and family members, are being accepted through March 2 via an online…

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…