News

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Free Regulatory Support Offered to Ultra-rare Disease Advocates

The clinical research organization MMS Holdings has launched a free program to help advance research and provide regulatory support to ultra-rare disease patient advocacy groups. Most rare diseases are ultra-rare conditions, according to a MMS press release, and require different approaches to navigating statutory requirements for drug development than more common…

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Virtual AADC Deficiency Awareness Day is Oct. 22

The second AADC Deficiency Awareness Day will take place virtually on Oct. 22. It is sponsored by the AADC Family Network and PTC Therapeutics, which is currently developing a gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency. PTC announced the event, which aims to raise awareness…