News

Topline data should be available toward the end of this year for a Phase 3 clinical trial testing B-VEC (beremagene geperpavec), a topical gene therapy for treating skin wounds in patients with dystrophic epidermolysis bullosa (DEB). Krystal Biotech, the therapy’s developer, announced that the last of the 31 participants in…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

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…

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 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…

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…

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.

A Phase 2 trial into the safety and early efficacy of INM-755, a cannabinol cream for people with epidermolysis bullosa (EB), will soon begin enrolling patients across Europe and Israel. “The start of this Phase 2 clinical trial represents a very important step forward to test the efficacy of INM-755…

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…

HMP Global’s Symposium on Advanced Wound Care (SAWC) Fall has established partnerships with the Wound Healing Foundation and debra of America to boost their research and improve care of patients with chronic wounds, such as those with epidermolysis bullosa (EB). The SAWC Fall is scheduled to…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

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…