Regulators

FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

Click here for the press release.

FDA´s DMD Draft Guidance

The FDA issued a guidance document. Here you will find the links to the materials for FDA’s DMD Draft Guidance: the Federal Register notice, the DMD Draft Guidance, and the CDER Statement.

Video of the exon skipping workshop at the EMA, Wednesday April 29th 2015

Please click here to watch the video of the workshop on Antisense oligonucleotide-mediated exon skipping therapy development for Duchenne muscular dystrophy (DMD).

EMA Guideline on the quality, non-clinical and clinical aspects 4 of gene therapy medicinal products

Please click here for the European Medicines Agency guideline.

EMA recommends first-in-class medicine for treatment of Duchenne muscular dystrophy

Click here for the European Medicines Agency recommendation.

« Page 1 »