Positive Results from Phase 3 Study of Acute Hepatic Porphyria Drug

Earlier this week, a press release from Alnylam Pharmaceuticals, Inc, announced the that the phase 3 study of their experimental acute hepatic porphyria drug Givosiran yielded positive results. According to the…

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Investigational Myelofibrosis Drug Granted Priority Review by FDA

A publication from OncLive recently reported that the FDA has granted the experimental myelofibrosis drug Fedratinib a priority review designation, following Celgene Corporation's submission of a New Drug Application (NDA) to the…

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Experimental MPS VI Drug Granted Rare Pediatric Disease Designation

A recent press release from the French biopharmaceutical company Inventiva announced that the Food and Drug Administration (FDA) had granted the company's experimental mucopolysaccharidosis VI (MPS VI) drug odiparcil rare…

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Scientists are Ramping Up Their Attack on Alzheimer’s Disease Through Immunotherapy

In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…

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Against the Odds: Maria’s Journey with – and beating – Rare Epithelioid Sarcoma

Wisconsin; the land of beer, cheese, good-hearted people, and Maria Voermans - rare cancer survivor and now fierce advocate for the rare disease community.  Ten years ago, Maria had the…

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