News FDA probing deaths with DMD gene therapy Elevidys The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.
News DMD gene therapy Elevidys linked to new liver failure case Another death from liver failure has been seen with Roche and Sarepta's Duchenne muscular dystrophy therapy Elevidys, prompting a pause on treatment.
News Italfarmaco closes on EU okay for Duchenne drug Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD).
News Multiple studies of Sarepta's DMD therapy halted after death Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.
News Sarepta slumps on Elevidys patient death The death of a patient treated with Sarepta's Duchenne muscular dystrophy gene therapy Elevidys has hit its share price.
News Santhera's DMD drug poised for wider rollout in Germany Santhera has agreed pricing and reimbursement for its Duchenne muscular dystrophy drug Agamree with German health funds representing 90% of patients.
Market Access The value of a partner, globally and locally A new podcast looks at recent trends related to drug approvals, as well as challenges and opportunities both small and large pharma companies face