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 lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

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…

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, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

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. 20 webinar.  William Whitman…

The rare case of a man infected with the hepatitis E virus who developed autoimmune hemolytic anemia was described in a study that also highlighted the potential for underdiagnosing autoimmune complications in such infections. The study, “Unusual Presentation of Hepatitis E with Autoimmune Hemolytic Anemia – A Case Report,”…

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 the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Autoimmune hemolytic anemia (AIHA) experts gathered in Vienna, Austria, in November 2017 for the First International Consensus Meeting with the goal of summarizing current research into diagnosis and treatment of the rare genetic disorders. Now, after three subsequent meetings at medical conferences, the researchers and physicians have published their conclusions and…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Physicians have described the rare case of a woman with cold agglutinin disease (CAD) associated with advanced breast cancer. Her case was described in a case report study, “Cold autoimmune hemolytic anemia: a rare association with triple-positive breast cancer,” published in the Journal of…