Catalyst Pharmaceuticals Announces Support of Rare Disease Day 2019
“We are proud to support Rare Disease Day and recognize the importance of investment in medical research that is needed to improve the lives of patients who are afflicted with rare and ultra-rare diseases,” said
“To be able to dance with my daughter at her wedding was life-changing for me. With the recent approval of Firdapse I’m energized by the increased awareness, education and support that is now available for the entire LEMS community”, said
For information about Rare Disease Day activities, visit https://rarediseases.org/rare-disease-day/.
About Lambert-Eaton Myasthenic Syndrome (LEMS)
LEMS, is a rare autoimmune disorder, most often characterized by fatigable limb muscle weakness. The disease is caused by autoantibodies against voltage-gated calcium channels located in the nerve-muscle junction, resulting in improper nerve-muscle communication, leading to progressive muscle weakness, when left untreated. In approximately 50% of cases, LEMS is associated with an underlying malignancy, most commonly small cell lung cancer, and in some individuals, LEMS is the first symptom of such malignancy. LEMS generally affects the extremities, especially the legs. In early course of the disease, muscles closest to the trunk are affected, which are manifested as difficulties with climbing stairs or rising from a sitting position. Physical exercise and high temperatures tend to worsen the symptoms. Other symptoms occasionally seen include weakness of the muscles of the mouth, throat, and eyes. Individuals affected with LEMS also may have a disruption of the autonomic nervous system, with symptoms of dry mouth, dry skin, constipation, blurred vision, impaired sweating, and/or inappropriate blood pressure variations.
Catalyst Pharmaceutical is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), MuSK antibody positive myasthenia gravis (MuSK-MG), and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was recently approved by the
Firdapse is currently being evaluated in clinical trials for the treatment of CMS, MuSK-MG and SMA Type 3 and has received Orphan Drug Designation from the
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Catalyst will be successful in commercializing Firdapse, (ii) whether, even if Catalyst is successful in commercializing Firdapse, Catalyst will become profitable, (iii) whether Firdapse will ever be approved for the treatment of CMS, MuSK-MG, SMA Type 3, or any other disease, and (iv) those other factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2017 and its other filings with the
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Source: Catalyst Pharmaceuticals, Inc.