News

Corbus Pharmaceuticals has extended its intellectual property portfolio with a new patent fo its investigative cannabinoid-derived drug candidate anabasum to treat inflammatory diseases. The new patent (No. 9,801,849), issued by the U.S. Patent and Trademark Office, protects the use of anabasum for the treatment of systemic sclerosis, dermatomyositis,…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

Specific autoantibodies may give rise to clear differences in disease features among Spanish patients with scleroderma, according to a report in the journal Clinical and Experimental Rheumatology. These differences are similar to those reported in other populations, and suggest that analyses of specific autoantibodies may provide physicians with additional…

Male gender and certain lung function parameters are potential risk factors for the progression of pulmonary arterial hypertension in patients with systemic sclerosis-PAH (SSc-PAH), an analysis of the DETECT study shows. The research, “Factors associated with disease progression in early-diagnosed pulmonary arterial hypertension associated with systemic sclerosis: longitudinal data from…

Emerald Health Pharmaceuticals, which develops synthetic medications based on cannabinoid science, has received orphan drug status from the U.S. Food and Drug Administration (FDA) for its lead molecule, EHP-101, to treat systemic scleroderma (SSc). EHP-101, which also has the potential to treat multiple sclerosis (MS), is one of…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

Nintedanib alleviated the core manifestations of systemic sclerosis, resulting in a marked improvement in the disease, a study in mice has shown. The research, published in the Annals of the Rheumatic Diseases, was titled “Nintedanib inhibits macrophage activation and ameliorates vascular and fibrotic manifestations in the…

New mutations found in the GNPTG gene were identified as the underlying cause of a familial form of a scleroderma-like disease, according to a study published in the journal Pediatric Rheumatology. The study, “Next Generation Sequencing identifies mutations in GNPTG gene as a cause of familial form…

A new treatment using adipose-derived regenerative cells (ADRCs) to treat hand problems caused by scleroderma is now available at Okyanos Center for Regenerative Medicine in Freeport, Grand Bahama. Researchers developed the treatment protocol based on the STAR clinical study sponsored by Cytori Therapeutics. “Through the delivery of regenerative…