Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease. This treatment has also received…
According to a story from globenewswire.com, the biotechnology company Emerald Health Pharmaceuticals Inc., has announced recently that its experimental drug EHP-102 has earned Orphan Drug designation from the European Medicines…
According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…
FightMND is an Australian nonprofit organization dedicated to improving the lives of motor neurone disease patients through funding research for cures. They've successfully provided millions toward research initiatives and clinical…
Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…
AI Therapeutics is a biotechnology company that uses artificial intelligence to match drugs to indications. They do so through the use of their algorithm, Guardian Angel. This algorithm has…
Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
Oliver Sacks is beloved for his best-selling books that detail case studies of rare neurological disorders. Sacks created a niche when he combined his work as a neurologist with whimsical…
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