This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in developing treatments for rare diseases, and new findings about Zika virus-associated Guillain-Barre syndrome.
The FDA Grants Approval to Begin Dosing First-in-Human CRISPR Technology to Treat Duchenne Muscular Dystrophy
Could CRISPR gene-editing help achieve a breakthrough for Duchenne muscular dystrophy? A new trial is about to begin.
Recent research has revealed the Guillain-Barre syndrome caused by infection with the Zika virus appears to result in reduced sense of smell.
Parents of patients with rare diseases play a central role in spearheading rare disease research and drug development.