U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME
"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
- "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
- The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
- When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
- "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.