News

People with acute hepatic porphyria (AHP) are more likely to file for long-term sick leave and disability pension, but are not at more risk of premature death than the general population, a nationwide study from Norway found. The study, “Sick leave, disability, and mortality in acute…

Alnylam Pharmaceuticals is not expecting the production and supply of Givlaari (givosiran), a treatment for acute hepatic porphyria (AHP), to be affected by the COVID-19 pandemic, the company said. Besides Givlaari, Alnylam also markets Onpattro (patisiran), which is approved in the U.S. for treating …

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Physicians solved a case of porphyria cutanea tarda (PCT) — with blistering lesions on the patient’s arms, neck and face — by treating an underlying infection caused by the hepatitis C virus (HCV), according to a new report. The case was described in the study, “…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

To help patients and families facing out-of-pocket medical costs, The Assistance Fund (TAF) has opened a new program for eligible individuals living with porphyria. The TAF Porphyria Financial Assistance Program helps with therapy-related copayments, health insurance premiums, and incidental medical expenses related to the metabolic disorder, which has…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The European Commission has approved Givlaari (givosiran) to treat acute hepatic porphyria in patients, 12 and older, in the European Union. This follows Givlaari’s approval for the same indication in the U.S., and the positive recommendation recently issued by the Committee for Medicinal…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…