ProQR Therapeutics is partnering with the nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF)…
Articles by José Lopes, PhD
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BERG’s BPM 31510 (ubidecarenone), a…
Results from a Phase 1/2 trial in recessive dystrophic epidermolysis bullosa (RDEB) showed that gene therapy candidate EB-101…
The U.S. Food and Drug Administration granted rare pediatric disease designation to Castle Creek Pharmaceuticals’ diacerein 1% ointment…
The U.S. Department of Defense awarded $3.8 million to a consortium working to develop stem cell-based therapies for patients with…
Krystal Biotech has submitted an investigational new drug application with the U.S. Food and Drug Administration (FDA) to start a Phase…
Netherlands-based ProQR Therapeutics is supporting Rare Disease Day 2018 and will celebrate the legacy of rare disease pioneer Henri…
Combining genetic and protein analyses is a successful strategy to identify unusual cases of epidermolysis bullosa (EB), a German…
The U.S. Food and Drug Administration (FDA) will allow pediatric patients to enroll in the Phase 2 segment of an ongoing…
Patients with dystrophic epidermolysis bullosa (DEB) show increased pain, but no difference in anxiety or depression, in comparison with healthy…