Chiesi Global Rare Diseases is launching a research grant initiative to support innovative studies into Fabry disease, alpha-mannosidosis, and cystinosis — all three of which are lysosomal storage disorders (LSDs). “Whilst the disorders can be rare individually, their prevalence is significant at a global level with an estimated…

Fabry disease is a progressive, potentially life-threatening, rare, genetic disorder. Many people with Fabry disease succumb to heart disease, kidney disease, and stroke at relatively young ages. A common question asked by people diagnosed with Fabry disease is, “How long will I live?” When I first learned that I have…

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…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

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…