News

March 31, 2020April 1, 2020

NORD Partners with ‘Sing Me a Story’ for July 18-20 Living Rare Forum

News

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 ... Read more

March 25, 2020March 25, 2020

Rubius Stops Clinical Development of RTX-134 Cell Therapy for Phenylketonuria

News

Rubius Therapeutics is discontinuing its Phase 1b trial investigating RTX-134, a potential cell therapy for people with phenylketonuria (PKU). The decision comes as the company shifts its focus to the ... Read more

March 23, 2020March 23, 2020

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

Just Published, 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 18, 2020March 18, 2020

Efficacy of Gene Therapy for PKU, HMI-102, Supported by Work in Mice

News

A single dose of HMI-102 — a candidate gene therapy for phenylketonuria (PKU) by Homology Medicines — lowered blood levels of phenylalanine and kept this amino acid‘s levels within a normal range over 48 weeks ... 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 lacking ... 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 #WhatMakesMeRare ... Read more

March 4, 2020March 4, 2020

Nutritional Counseling Can Help Keep Phe Levels Low in Teens, Young Adults

News

Professional dietary counseling by a nutritionist to young adults with phenylketonuria (PKU) has clear beneficial short-term effects on phenylalanine levels in blood, a study found. But nutritional counseling should be done ... Read more

March 2, 2020March 2, 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 26, 2020February 26, 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 26, 2020February 27, 2020

Highest Rates of PKU Found in Newborns in Turkey and Iran, Study Reports

News

Worldwide, phenylketonuria (PKU) is seen to affect about 6 of every 100,000 babies born, with the highest frequency being reported in Turkey and Iran, according to a review study based on newborn screening data. ... Read more