Happy Thursday!
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.
Study of the Week: Zika Virus-Related Guillain-Barre Syndrome Linked to Worsened Olfactory Function
Recent research has revealed the Guillain-Barre syndrome caused by infection with the Zika virus appears to result in reduced sense of smell.
Parents Develop Drugs to Help Their Children with Rare Diseases
Parents of patients with rare diseases play a central role in spearheading rare disease research and drug development.
