Catalyst Pharma's Firdapse bet on congenital myasthenic syndromes turns sour

Catalyst Pharma's Firdapse bet on congenital myasthenic syndromes turns sour

Source: 
Endpoints
snippet: 

Months after Firdapse maker Catalyst Pharmaceuticals filed a lawsuit against the FDA, the Florida company on Wednesday said the drug had failed a pivotal trial in patients afflicted with congenital myasthenic syndromes (CMS), an umbrella term for rare neuromuscular disorders comprising a spectrum of more than 50 genetic defects.