Rare Disease Day Celebrated at Virtual Nasdaq Opening Bell Ceremony

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by Patricia Inacio PhD |

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Catalyst Pharmaceuticals kicked off this year’s Rare Disease Day, along with rare disease advocacy partners, with a virtual opening bell ceremony at the Nasdaq stock market.

“Catalyst Pharmaceuticals and our dedicated team of employees, patients, and numerous patient advocacy groups are thrilled to be here at Nasdaq today for the opening bell ceremony and to celebrate on this great platform global Rare Disease Day,” Patrick J. McEnany, chairman and CEO of Catalyst Pharmaceuticals, said during the ceremony.

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Since it started in 2008, Rare Disease Day — celebrated on the last day of February — brings together patients, caregivers, friends, and advocates from around the world to raise awareness about rare diseases, including Lambert-Eaton myasthenic syndrome (LEMS).

“We are committed to making a meaningful impact on the lives of those living with rare diseases,” McEnany said in a press release. “Catalyst is working tirelessly with patients living with the rare condition Lambert-Eaton myasthenic syndrome (LEMS), helping them gain access to treatment.”

As part of these efforts, the company is working to facilitate diagnosis, “identifying appropriate treatment options, securing affordable access and offering resources to patients and families on their journey towards an FDA-approved safe and effective therapy,” McEnany said during the livestream.

Catalyst, which markets Firdapse (amifampridine), an approved treatment for adults with LEMS, organized the ceremony with participation from the National Organization for Rare Disorders (NORD), Global Genes, the Muscular Dystrophy Association, the Myasthenia Gravis Foundation of America, Conquer Myasthenia Gravis, and the Myasthenia Gravis Association.

“We thank our advocacy partners who are standing with us, echoing our relentless commitment to this cause,” McEnany said. “We are grateful to Nasdaq for the opening bell-ringing opportunity, allowing us to show our support for the rare disease community.”

While there are more than 7,000 recognized rare diseases worldwide, 5% have an approved treatment. The Nasdaq ceremony is part of efforts by pharma and patient advocacy groups to foster collaborations and speed the research and development of new treatments for rare disease patients.

“Rare Disease Day is an opportune moment to build awareness, consider our progress, and rally around what has yet to be done,” said Craig Martin, CEO of Global Genes. “Until the day rare disease becomes a global health priority, we must be relentlessly nimble, creative, resourceful, and collaborative in our efforts to improve the lives and care of the nearly one in 10 Americans and 400 million people worldwide living with rare conditions.”

The bell ringing ceremony at Nasdaq MarketSite is a great opportunity to showcase Rare Disease Day to a global audience, raising awareness and connect rare disease patients with those at the forefront of therapeutic development, advocates said.

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“Rare Disease Day and events such as this help individuals and families living with rare diseases know that they are not alone and that there is a community raising awareness on their behalf,” said Lisa Sarfaty, vice president of community engagement at NORD.

“NORD is grateful for this opportunity to champion health equity for the rare disease community and help shine a light on their unique experiences and challenge,” she added.

A replay of the virtual ceremony is available here.