Alnylam Pharmaceuticals has awarded a total of $270,000 to seven patient advocacy groups, including the American Porphyria Foundation (APF), in the…
Articles by Patricia Inácio, PhD
A Phase 3 clinical trial of Mitsubishi Tanabe’s investigational therapy MT-7117 (dersimelagon) for people with erythropoietic protoporphyria (EPP) or…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Patients with acute intermittent porphyria (AIP) who take hemin as preventive therapy use fewer healthcare resources than those…
A tissue-specific mechanism was found to take part in the regulation of iron levels in the body, a discovery that…
Most acute hepatic porphyria (AHP) patients with recurrent attacks also experience significant chronic symptoms between attacks, with more than 75%…
Toxic accumulation of protoporphyrin IX in the skin and liver of people with erythropoietic protoporphyria (EPP) — the most common…
The U.S. Food and Drug Administration (FDA) has accepted and granted priority review status to Alnylam Pharmaceutical’s new drug…
Researchers identified two previously unknown mutations in the coproporphyrinogen oxidase (CPOX) gene as the underlying cause of a rare form…
Alnylam Pharmaceuticals has announced that its investigational therapy givosiran achieved the primary efficacy and the majority of secondary endpoints in…