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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…

Urinary symptoms are frequent in patients with systemic sclerosis (SSc), especially in patients with limited cutaneous SSc, which have a 2.2 times higher risk of developing urinary symptoms than other SSc patients, and in patients who are positive for anti-centromere antibodies, who have a 2.8 times increased risk. Findings…

The innate immune system and small RNA molecules known as microRNA are key factors in the development of scleroderma, according to a study based on previously published research. The study, “Inflammasome lights up in systemic sclerosis,” was published in the journal Arthritis Research & Therapy. The…

Viracept (nelfinavir mesylate, NFV), an antiretroviral drug approved as a therapy against the human immunodeficiency virus (HIV), could be beneficial in the treatment of systemic sclerosis (SSc), a study on a mouse model of the disease shows. The study, “The Antiretroviral nelfinavir mesylate, a potential therapy for systemic scleroderma,”…

Scleroderma Canada and the Pulmonary Hypertension Association of Canada have collected more than 2,000 letters signed by pulmonary arterial hypertension (PAH) patients, their caregivers and loved ones urging politicians to make all PAH treatments accessible as soon as possible through public funding. Health Canada approved Actelion’s Uptravi (selexipag) in January…