News

March 25, 2020March 25, 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 the ... Read more

March 10, 2020March 10, 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 lacking ... Read more

March 5, 2020March 6, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News, uncategorized

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 ... 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, and ... Read more

February 27, 2020February 27, 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. 20 ... Read more

February 20, 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 the ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

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, ... Read more

February 7, 2020February 7, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

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 ... Read more

January 2, 2020January 2, 2020

EPOCH Combo Therapy Enhances Survival for Some Patients with Non-Hodgkin’s Lymphoma-associated HLH, Phase 2 Study Shows

News

Dose-adjusted EPOCH — in combination with rituximab — shows promising results as a first-line therapy for hemophagocytic lymphohistiocytosis (HLH) in people with B-cell non-Hodgkin’s lymphoma (NHL), a Phase 2 trial ... Read more

December 19, 2019December 19, 2019

Newly ID’d Mutation Linked to HLH Symptoms of Chronic Inflammation in Teen, Study Reports

News

A previously unknown mutation in a protein that regulates immune activation, called Roquin-1, was found to be the underlying cause of uncontrolled cytokine release and excessive inflammation — hallmarks of ... Read more