News

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

A gene expression analysis identified several deregulated pathways in fibroblasts from classical Ehlers-Danlos syndrome (EDS) patients, offering new insights for future studies investigating the disease mechanisms and potential therapies, a study reports. The study, “Molecular insights in the pathogenesis of classical Ehlers-Danlos syndrome from transcriptome-wide expression profiling of…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

Erector spinae plane (ESP) block can improve the safety of surgical procedures in patients with Ehlers-Danlos syndrome (EDS) and offer effective pain relief, according to a case report. The study evaluated pain perception in a patient after vascular ring repair surgery. The case report, “The erector spinae plane block…

The U.S. Food and Drug Administration (FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history — how disorders such as spinal muscle atrophy (SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical…

The Cherry Hill Education Association raised more than $10,000 for the non-profit organization Ehlers-Danlos Society through its 14th annual “Volley for Support” fundraising tournament that took place in October 2018. The Society’s global community includes patients, caregivers, healthcare professionals, and supporters, who work to improve the lives of…

Patients diagnosed with periodontal Ehlers-Danlos syndrome (EDS) may also have changes in the brain’s white matter, which makes up the tissue in the brain and spinal cord, a case series reports. Periodontal EDS is the rarest form of EDS, and is characterized by severe early-onset gum disease. The…

Generex Biotechnology will launch a wound care gel formulation called Excellagen, a potential treatment option for patients with Ehlers-Danlos syndrome (EDS) among others disorders. Excellagen is a highly-purified type 1 collagen-based, topical gel. According to Generex, its 3D dermal matrix favors wound healing. It activates platelets, leading to the…

Edsivo (celiprolol), being developed by Acer Therapeutics, is one step closer to approval for the treatment of vascular Ehlers-Danlos syndrome (vEDS), as the U.S. Food and Drug Administration (FDA) agreed to review the new drug application (NDA) for the therapy. The regulatory agency also granted priority…