News

March 20, 2020March 20, 2020

Porphyria Cutanea Tarda Solved by Treating HCV Infection, Study Reports

News

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 ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

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 ... Read more

March 13, 2020March 13, 2020

The Assistance Fund Launches Aid Program for Out-of-pocket Medical Costs

News

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 ... Read more

March 12, 2020March 12, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

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 ... Read more

March 6, 2020March 6, 2020

Givlaari Approved in EU for Patients With Acute Hepatic Porphyria

News

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 ... Read more

March 5, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News

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 ... Read more

March 3, 2020March 3, 2020

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

News

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more

February 28, 2020February 28, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

News

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. ... Read more

February 21, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 14, 2020February 14, 2020

AIP Patients Use Fewer Healthcare Resources When They Take Hemin as Preventive Therapy, US Study Reports

News

Patients with acute intermittent porphyria (AIP) who take hemin as preventive therapy use fewer healthcare resources than those who take the medicine for acute attacks, according to a study ... Read more

Pin It on Pinterest