Acute hepatic porphyria, or AHP, is a group of rare genetic conditions that affect how the body makes a natural substance called heme. Heme is produced in the liver and is essential for many important ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company and its collaborators will present at the 2019 ...

Please provide your email address to receive an email when new articles are posted on . LONDON — Long-term givosiran treatment provided sustained benefit and improved quality of life among patients ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today on Rare Disease Day announced the premiere of its new documentary, Two of ...

Up-regulation of hepatic delta-aminolevulinic acid synthase 1 (ALAS1), with resultant accumulation of delta-aminolevulinic acid (ALA) and porphobilinogen, is central to the pathogenesis of acute ...

The program, called Alnylam Act ®, provides genetic testing for the diagnosis of acute hepatic porphyria, primary hyperoxaluria type 1 (PH1), and hereditary ATTR (hATTR) amyloidosis. Along with ...

The acute hepatic porphyrias (AHP) are rare disorders that typically become apparent during adulthood and may have varied presentations. Most of these quiz questions are derived from recent literature ...

WEST DES MOINES, Iowa — Feb. 29, the rarest day of the year, marks the celebration of Rare Disease Day. The goal of the day is to raise awareness and support as in the United States, one in 10 people ...