Congenital adrenal hyperplasia is a rare, severe, and lifelong genetic disease of the adrenal gland that impairs its ability to produce sufficient cortisol and leads to excessive production of androgens, resulting in hormonal imbalances. It is reported that an innovative drug has recently been introduc...
Rare Disease Innovation at the FDA—Opportunities for Implementation Carolyn K. Shore, PhD1; Tequam L. Worku, MPH1; Jeffrey Kahn, PhD, MPH2 Author Affiliations JAMA. 2024;332(23):1973-1974. doi:10.1001/jama.2024.20358 Full Text Over the past 4 decades since the Orphan Drug Act was...
Rare Disease Linked to MS Drug Worries FDA
An application can provide a list of specialists, genetic testing, remote self-testing, and continuous telemonitoring of health status, all of which could help lower the TTD of RDs and lower disease burden from this community.22 Role of Artificial Intelligence and Medical Imaging Recent ...
FDA grants rare pediatric disease designation to incentivize the development of new treatments for serious and life-threatening diseases that primarily affect children aged 18 years or younger, with fewer than 200,000 ...
The FDA approved IntraBio Inc.'s Aqneursa (levacetylleucine) yesterday for the treatment of ultra rare neurodegenerative disease Niemann-Pick Disease, Type C (NPC), according to a news release. This makes Aqneursa the second NPC drug approved within a week and the first standalone FDA...
The FDA has granted the novel GSK-3β inhibitor elraglusib rare pediatric disease designation for the potential treatment of patients with Ewing sarcoma.1 “Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with Ewing sarcoma...
The FDA has granted a rare pediatric disease designation to GPS for the treatment of pediatric patients with AML.1 "GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients,” said Angelos Stergiou, MD, ScD hc, president...
Ionis Pharmaceuticals on Thursday won Food and Drug Administration approval of a therapy for patients with a rare and deadly genetic disease that impedes the body’s ability to break down fats, setting the stage for the company to kick off the first solo drug launch in its 35-year hi...
Chairman and CEO of Moleculin said. "It is also important for investors to understand the implications of the PRVs or Priority Review Vouchers. These vouchers are issued upon drug approval of the rare disease indication from the FDA and, once issued, can be transferr...