It has been a busy time in the Duchenne arena. In the US, the FDA has granted accelerated approval for the gene therapy treatment SRP-9001.
This is the first time a gene therapy for Duchenne has been approved anywhere in the world, and represents a very exciting step forward for the Duchenne community. However this approval only applies in the US, and the treatment will only be available to patients who meet the required criteria.
SRP-9001 has been developed by Sarepta Therapeutics.
Many families will understandably want to know if and when the treatment will be approved in the UK. Roche, which holds the licence for SRP-9001 in the UK, has shared a letter to the community explaining its progress in engaging with the MHRA – the UK’s drug approval body.