Library

Campbell E, Ross LF. Parental attitudes regarding newborn screening of PKU and DMD. American journal of medical genetics. Part A…

Parsons EP, Clarke AJ, Hood K, Lycett E, Bradley DM. Newborn screening for Duchenne muscular dystrophy: a psychosocial study. Archives…

The FDA issued a guidance document. Here you will find the links to the materials for FDA’s DMD Draft Guidance:…

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

Please click here for the European Medicines Agency guideline.

Buyse GM, Voit T, Schara U, et al. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy…

Click here for the European Medicines Agency recommendation.