Andrea Lobo, PhD, science writer —

Catalyst Pharmaceuticals will mark this year’s Rare Disease Day by ringing the closing bell Feb. 28 at the Nasdaq Stock Market. The ceremony will begin at 3:30 p.m. ET, and can be viewed via livestream. Rare Disease Day, celebrated annually on the last day of February, raises awareness about rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS), by bringing together patients, caregivers, and advocates worldwide. The event aims to recognize those who work to develop new treatments for patients living with rare diseases. Last year, Catalyst participated, together with rare disease advocacy partners, in the event’s opening bell ceremony. “We are honored to stand with the rare disease communities on International Rare Disease Day to help heighten the important need for new and innovative medicines for the treatment of rare diseases,” said Patrick J. McEnany, chairman and CEO of Catalyst, in a press release. “Catalyst recognizes the valuable partnerships with leading advocacy groups who share in the mission of raising awareness for education, diagnosis, and treatment access. We thank Nasdaq for their continued support and all our valued advocacy groups for their steadfast commitment to the rare disease community we all serve.” LEMS is a rare autoimmune disease in which the immune system mistakenly launches an attack against specific calcium channels found in nerve cells that are key in controlling muscle movements. Its symptoms include muscle weakness that often starts in the legs, making it difficult for patients to walk and climb stairs. As the disease progresses, weakness may spread to other muscles, affecting everyday tasks. “People newly diagnosed with an ultra-rare disease like Lambert-Eaton Myasthenic Syndrome often began their journey being undiagnosed or misdiagnosed for years due to a lack of awareness and knowledge,” said Price Wooldridge, board president of The Lambert-Eaton LEMS Family Association. Catalyst markets Firdapse (amifampridine), an oral therapy for LEMS, that’s intended to help enhance muscle strength. Initially approved in 2018 by the U.S. Food and Drug Administration for patients ages 17 and older, its use was expanded in 2022 to include children as young as 6. “It’s highly visible initiatives like these that raise awareness and inspire discussions that may offer patients and their caregivers hope for earlier diagnosis and new, innovative treatments. We appreciate this opportunity to unite in this cause,” Wooldridge said. The bell-ringing ceremony is an opportunity to expose Rare Disease Day to a global audience, raising awareness and connecting patients with those at the leading edge of treatment development. “Today, rare diseases affect over 25 million Americans. While important progress has been made over the years, there remains a significant urgency to address the critical unmet needs of these individuals,” said Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders. “We are pleased to partner with Catalyst on Rare Disease Day. Through our shared commitment, we are raising awareness on the importance of patient advocacy, new treatments, and support for individuals and families living with rare diseases.”

Andrea Lobo, PhD, science writer —

Articles by Andrea Lobo

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