Apellis Pharmaceuticals announced that the European Medicines Agency granted orphan drug designation to APL-2 in the treatment of PNH patients.
AMC creates medicines for children afflicted with diseases which don’t present a business opportunity for the extant commercial pharmaceutical industry.
In the latest issue of the New England Journal Medicine, there is a report on the success of Bluebird Bio’s gene therapy for sickle cell disease.
Penn Medicine's Orphan Disease Center (ODC) is partnering with FAST for gene therapy research.
The App was developed by Misha Rosenbach, MD and Daniel O’Connor, a fourth-year medical student at University of Pennsylvania.
Results from a Phase 1 trial showed encouraging activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas.
The European Medicines Agency (EMA) validated the previously submitted Marketing Authorization application (MAA) for eteplirsen to treat Duchenne muscular dystrophy amenable to exon 51 skipping.
A "zombie outbreak" occurred in New York over the summer due to a synthetic cannabinoid drug.
A new study published in the New England Journal of Medicine questions the ability of whole genome sequencing to be the best means to diagnose patients.
The resulting cell has the mother’s nuclear DNA but mostly the donor’s normal mtDNA. Add sperm and what results is a fertilized ovum with healthy mtDNA.
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