Editor’s Choice: A New CRISPR Trial, Revelations About Zika-Related GBS, and Parents Taking Action

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.

source: pixabay.com

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.

 

 

 

source: shutterstock.com

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.

source: shutterstock

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.

 

 

 


Do you have a rare disease experience of your own? Share with us here.

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