Variegate porphyria (VP) is an autosomal dominant condition that results from the deficiency of protoporphyrinogen oxidase (PPOX, EC 1.3). The build-up of porphyrin precursors delta-aminolevulinic ...

Porphyria cutanea tarda (PCT) is a common form of hepatic porphyria characterized by symptoms such as fragile, blister-prone skin particularly on sun-exposed areas. The condition occurs due to an ...

Please provide your email address to receive an email when new articles are posted on . Clinicians are hoping an RNA interference therapeutic targeting aminolevulinic acid synthase 1, known as ...

Acute porphyria is a group of uncommon diseases that can cause severe, potentially life-threatening attacks of abdominal pain, nausea, vomiting and paralysis. Liver transplantation is currently the ...

Givlaari (givosiran) is a prescription drug approved to treat acute hepatic porphyria (AHP). Givlaari comes as an injection given under the skin by a healthcare provider. Specifically, Givlaari is ...

DEAR DR. GOTT: I am a concerned grandmother writing about my 11-year-old granddaughter. She was diagnosed with porphyria at the age of two. Her hands and feet start hurting so badly that she has to ...

The FDA has approved a new treatment for adult patients with acute hepatic porphyria (AHP) a rare genetic disorder. Givlaari is an RNA interference therapeutic targeting aminolevulinic acid synthase 1 ...

About Givosiran Alnylam is developing givosiran (formerly known as ALN-AS1), a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the ...

BOSTON--Dana-Farber Cancer Institute researchers say they have uncovered a molecular explanation for the episodic attacks of irrational and demented behavior in porphyria, the disease believed to have ...

ORLANDO, Fla., Feb. 19, 2020 /PRNewswire/ -- The Assistance Fund (TAF), an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs ...