News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

Implanting some of scleroderma patients’ fat cells in their fingers significantly improved their hand function and overall disability, a clinical trial showed. The STAR trial (NCT02396238) dealt with Cytori Therapeutics’ Habeo cell therapy. It involved placing fat — or adipose — cells under the skin of each finger. Cytori presented…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

Boehringer Ingelheim and the Scleroderma  Foundation have started a global initiative to raise awareness of scleroderma and support those with the life-threatening disease. In conjunction with the initiative, which is dubbed More Than Scleroderma: The Inside Story, they created a website that will feature the latest scientific knowledge and news on…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

The European Medicines Agency has granted orphan drug designation to Emerald Health Pharmaceuticals’  lead cannabis-based product for treating systemic scleroderma. EHP-101 is derived from cannabidiol, a chemical found in the cannabis plant. The therapy activates the cell-surface receptors PPARγ and CB2, which in turn activate cell signaling pathways that can…

The National Institutes of Health’s (NIH) National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) recently awarded five new Core Centers for Clinical Research (CCCR), one of them with a particular focus on scleroderma. The goal of the CCCRs is to develop and apply methods that…

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…

Immunosuppressive therapy followed by an autologous stem cell transplant resulted in significant long-term benefits, including improved overall survival, in adult scleroderma patients compared to immunosuppressive therapy alone, according to the results of a Phase 2/3 trial. Despite a higher rate of treatment-related deaths in the transplant group, researchers argue…

Fibrocell Science has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for FCX-013, its gene therapy candidate for moderate to severe localized scleroderma. An IND is a request for FDA authorization to administer an investigational therapy to humans. It is generally…