Catalyst Pharmaceuticals Supports Rare Disease Day® and Joins Global Movement to Raise Important Awareness for Rare Diseases

Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), (Catalyst) a biopharmaceutical company focused on  developing  and  commercializing  innovative  therapies  for  people  with  rare  debilitating diseases, today announced that  it  has  joined  forces  with  30  million  Americans  and  health  care  advocates around the world for Rare Disease Day® on February 28. Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges they present to patients and families.
Catalyst continues to support patient organizations that provide disease awareness for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS). These are rare diseases in which patients are often misdiagnosed with other diseases prior to getting a definitive diagnosis, thus educating the patient and physicians at medical congresses is an important outreach to the rare disease community to facilitate proper and prompt diagnoses.”Completing one phase 3 clinical study and enrolling a second phase 3 clinical study for LEMS indicates our commitment to the LEMS community for patients to have access to an FDA approved treatment” says Patrick J. McEnany, Chairman and CEO of Catalyst. “In addition, we have expanded our CMS phase 3 clinical study to include adults in recognition of the need to provide access to a potential treatment across the entire CMS population. We are pleased to continue to support the efforts of the rare disease community to find ways to broaden the awareness of rare diseases and improve access to treatments.”According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and nearly half of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments.Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases and one which Catalyst actively supports.  Additionally, Catalyst supports Global Genes in their efforts to continue to get patients with rare diseases to take an active role in raising awareness about their disease.For more information about Rare Disease Day in the U.S., go to https://www.rarediseaseday.us.  For information about global activities, go to www.rarediseaseday.org. To search for information about rare diseases, visit NORD’s website www.rarediseases.org and Global Gene’s website https://globalgenes.org/world-rare-disease-day.
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