Phase 2b Trial Finds Firdapse Effective, Safe in Adults with MuSK-MG

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.

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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…