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Treatment of adult MuSK-antibody positive myasthenia gravis (MuSK-MG) patients with the investigational therapy Firdapse (amifampridine phosphate) improved muscle strength, eased fatigue, and was safe, according to full results of a Phase 2b clinical trial. Recruitment is ongoing for a phase 3 clinical trial (NCT03579966) testing Firdapse in MuSK-MG patients.

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Topline results from a Phase 2 trial of  Ra Pharma’s zilucoplan (previously known as RA101495), a potential self-administered therapy for patients with generalized myasthenia gravis (MG), showed a statistically significant decrease in markers of disease severity, achieving both its primary objectives. All the trial participants completed the 12-week treatment; 98 percent of…