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This month’s Plunge for Elodie will be a virtual event for a first time, with participants worldwide encouraged to plunge into freezing waters at their respective locations to raise funds in support of new therapies for epidermolysis bullosa (EB). …
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Children with epidermolysis bullosa (EB) who accept their condition and emotionally detach themselves from it tend to have a better quality of life than children who are very emotional and use other strategies to cope with their disease, a Dutch study has found. Likewise, when the parents of these…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
When injected into the bloodstream, mesenchymal stem cells (MSCs) — cells that can grow into different cell types and have potent regeneration properties — can temporarily reduce pain, skin itching and blistering, and improve the quality of life of children and adults with recessive dystrophic epidermolysis bullosa…
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
Infection by the bacteria Staphylococcus aureus is detected in most wounds of patients with epidermolysis bullosa (EB), according to an analysis of a U.S. and Canadian database. The study “Characterization of wound microbes in epidermolysis bullosa: Results from the epidermolysis bullosa clinical characterization and outcomes database” was…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
Abeona Therapeutics will proceed with enrollment for its pivotal Phase 3 study of EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB), following a meeting with the U.S. Food and Drug Administration (FDA). “We appreciate the clarity provided by the FDA and we are pleased to be…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
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