New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety.
SAE Media Group is delighted to announce the 2nd Annual Conference Oligonucleotide Therapeutics & Delivery, taking place on the 21st and 22nd September 2022 in London, UK.
The EMA's human medicines advisory committee has recommended that BioMarin Pharmaceutical's haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to
The safety of Astellas' gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845.
