TGIF, Patient Worthians!
This week, kids went to bed wearing inside out pajamas and wishing for snow. Their wishes came true– and so did the wishes of many rare disease families, praying for new treatments.
We have a story about a new FDA approval for MPS VII, promising research for Charcot-Marie-Tooth disease, and the continuation of testing for a Pompe disease treatment. We also have a contributor article discussing how rare parents can take care of themselves in stressful situations.
Sit back and enjoy this week’s Editor’s Choice.
The First Treatment in History for Mucopolysaccharidosis VII Was Just Approved by the FDA
Many families have been waiting so long for a treatment that could change the lives of their kid with MPS VII. For the first time ever, it’s here.
Read all about it here.
New Study Identifies Compounds that may Treat Mutations in Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth disease has stumped researchers for years. Could a cure be close?
Read more about the story here.
Report: Pharma Can Resume Testing Drug Efficacy for Neurological and Respiratory Conditions
A Pompe disease treatment that hit a snag in the road is going back into testing.
Read more about the story here.
How to Survive and Thrive: Tips From an LGS Mother
No one can deny that being a parent of a child with a rare disease is stressful- but thoughtful self-care can help you make the most of it.
Read the story here.
