News

Grant Supports Phase 3 Trial of FCX-007 for RDEB

The U.S. Food and Drug Administration (FDA) has awarded Castle Creek Biosciences a research grant to support its Phase 3 clinical trial of FCX-007 (dabocemagene autoficel), an investigational gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). The grant of $1.82 million, which comes from…

Celebrating Patient Achievements This National Awareness Week

From sharing stories to spreading the word about epidermolysis bullosa (EB) in person and on social media, supporters are poised to mark National Epidermolysis Bullosa Awareness Week, which runs annually in the U.S. from Oct. 25–31. According to the Dystrophic Epidermolysis Bullosa Research Association of America — coined as…

Global Genes, Diversity Coalition Team Up to Advance Health Equity

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

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…

Cannabinoids Ease EB Pain and Itchiness, Patients Say in Survey

Cannabinoid medications, those derived from cannabis, improve overall well-being and ease perceptions of pain and itchiness, more than 90% of epidermolysis bullosa (EB) patients who responded to a global survey reported. About 80% also reported taking fewer opioids, or stopping opioid use, to help with disease symptoms after starting on…

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…