News

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), said it will still take place at the…

The Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) has denied an appeal from Acer Therapeutics regarding Edsivo (celiprolol), an investigational therapy for vascular Ehlers-Danlos syndrome (vEDS). Further clinical studies will be needed before the investigational therapy can be resubmitted…

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) —…

The Ehlers-Danlos Society is offering a series of virtual events for community members to express concerns, ask questions, and address practical challenges during the global COVID-19 (Coronavirus) outbreak. The free, hour-long digital support meetings will run on the Zoom teleconferencing platform. Each chat session is limited to 100 people,…

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…

Adolescent girls with Ehlers-Danlos syndrome (EDS) often experience menstrual symptoms, but few are referred to gynecologic care, a small study suggests. Researchers emphasized that teenagers with EDS should receive early gynecologic counseling and treatment, since these problems can severely impact their well-being. These data also highlighted that most girls with…

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…