Catalyst Pharmaceuticals Announces Its Support of Spinal Muscular Atrophy Awareness Month
“We are proud to be part of the global movement to raise awareness of SMA and recognize the importance of developing new evidence-based medicines required to improve the lives of patients, families and caregivers afflicted with SMA,” said
“We are pleased to be leading the Catalyst sponsored pilot study of Firdapse® as a possible treatment option for SMA Type 3,” stated Dr.
Out of every 10,000 births, one baby is born with SMA. SMA is a genetic disease that affects the part of the nervous system that controls voluntary muscle movement. It is caused by malfunctioning genes that affect how nerve cells and muscle cells communicate across the neuromuscular junction—the place where nerve cells meet muscle cells. Patients with SMA can have severe muscle weakness in the upper arms, legs, and shoulders. In some instances, SMA may lead to paralysis or death.
Firdapse is being evaluated in clinical trials for the treatment of MuSK-MG, CMS, and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis and CMS. Firdapse (amifampridine) 10 mg tablets is the first and only approved drug in Europe for the symptomatic treatment in adults with LEMS.
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’s proof-of-concept study evaluating Firdapse for the treatment of SMA Type 3 will be successful, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2018 and its other filings with the
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Source: Catalyst Pharmaceuticals, Inc.