News

Rare Disease Day Events Bring Awareness, Equity to Patients

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 Plans to Request FDA Extend Use of Firdapse to Children

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…

Judgment in Favor of Catalyst Vacates FDA Approval of Ruzurgi

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…

NORD’s RareLaunch Platform Now Offering 2 New Courses

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…

Rare Disease Therapy Developers Saw Investment Jump 28% in 2021

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…

US Court Won’t Rehear Its Ruling Overturning Ruzurgi’s FDA Approval

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 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…