News

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FX-013, a Fibrocell gene therapy candidate to treat children with moderate to severe localized scleroderma. “Fibrocell CEO John Maslowski said in in a press release that the designation, in addition to FX-013’s Orphan Drug Designation,…

Soin Neuroscience recently submitted an orphan drug application to the U.S. Food and Drug Administration (FDA) for the company’s investigational drug TV1001SR (sodium nitrite), being developed for the treatment of scleroderma. Several completed clinical and preclinical trials have demonstrated TV1001SR enhances the growth of small arteries and promote blood flow. This…

Scientific advances have allowed researchers to develop therapies for pulmonary arterial hypertension (PAH) that have been shown to benefit patients with PAH related to systemic sclerosis (SSc). However, the median survival of these patients remains short, with a life expectancy of only four years from the time…

Long-term treatment with CellCept (mycophenolic acid) or Cytoxan (cyclophosphamide) can significantly reduce skin thickening in patients with systemic sclerosis (SSc), according to study published in the journal Arthritis Care & Research. About 90 percent of SSc patients have thickened skin — a hallmark of this disease. As such, it is commonly used…

Injecting bone marrow cells designed to produce high levels of thioredoxin 1 (Trx-1) restored skin tissue in mice with systemic sclerosis (SSc), according to Chinese researchers. Their study, “Bone Marrow-Derived Mesenchymal Stem Cells Expressing Thioredoxin 1 Attenuate Bleomycin-Induced Skin Fibrosis and Oxidative Stress in Scleroderma,” appeared in the…

Cytori Therapeutics recently completed all 48-week follow-up visits in its STAR trial evaluating adipose-derived regenerative cells (ADRCs) for the treatment of patients with scleroderma-caused hand dysfunction. A total of 88 participants were enrolled, and the last patient’s 48-week visit was conducted earlier this week, the company said in a press…

Recent data from the DeSScipher Project within the EUSTAR (European Scleroderma Trials and Research) group highlights the different perceptions on disability between patients with systemic sclerosis (SSc) and physicians, and identifies the main factors of disability perceived by SSc patients. The study, “Functional disability and its predictors in…

Results from a Phase 2 trial assessing the safety and efficacy of anabasum (formerly known as Resunab or JBT-101) shows promise in treating systemic sclerosis. That study is now being extended, and a Phase 3 trial is being planned. Anabasum, Corbus Pharmaceuticals’ lead candidate, is a synthetic oral drug that…

Systemic sclerosis (SSc) may be associated with changes in gut bacteria, according to a U.S.-Norwegian study which found that SSc patients are more prone to severe gastrointestinal tract (GIT) changes and symptoms. The study, “Systemic sclerosis is associated with specific alterations in gastrointestinal microbiota in two independent cohorts,” appeared in the…

A cell therapy based on injected genetically modified human skin cells and a pill that activates a protein has the capacity for breaking down fibrosis in localized scleroderma — at least in mice. Fibrocell and Intrexon will present preclinical data on their investigational cell therapy FCX-013 for the…