research

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

CHOC Children’s Given $8M to Develop Next-Generation Therapies for Pompe Disease

The Foundation of Caring has given $8 million to support CHOC Children’s efforts to develop next-generation therapies for Pompe disease. In honor of the gift, the program has been renamed the Foundation of Caring Lysosomal Storage Disorder Program at CHOC Children’s. The comprehensive multidisciplinary program…

European Initiative Targets Diagnosis, Treatment of Rare Diseases

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…