Tagraxofusp, a medicine approved in the U.S. under the name Elzonris to treat a rare blood cancer, may help decrease levels of immune cells that promote inflammation in systemic sclerosis (SSc), new preclinical data suggest. The results were shared in a poster, “CD123+ plasmacytoid dendritic cells (pDCs) from…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Injections of hyaluronidase, marketed as Hylenex, among other names, improved the ability to eat and reduced difficulties in opening the mouth and dental care in a woman with scleroderma, according to a new case report. The case was described in a study, “Hyaluronidase Treatment of…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
Treatment with pirfenidone fails to improve lung function in patients with systemic sclerosis (SSc)-associated interstitial lung disease (ILD), results of a clinical trial show. Trial results were discussed at the 2019 Annual European Congress of Rheumatology (EULAR) in Madrid, during an oral presentation titled “…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
Higher blood viscosity may help clinicians identify patients with systemic sclerosis at risk of having pulmonary arterial hypertension (PAH), a serious life-threatening respiratory complication. The finding was reported by Turkish researchers in the study “Whole blood viscosity in systemic sclerosis: a potential biomarker of pulmonary hypertension?”…
People with diffuse cutaneous systemic sclerosis (dcSSc) treated with lenabasum keep improving after 21 months of treatment, results from a Phase 2 open-label extension study show. Robert Spiera, MD, from Weill Cornell Medical College, New York, and principal investigator of the Phase 2 study, shared the data in…
Older men with diffuse cutaneous systemic scleroderma (dcSSc) have more estradiol, a form of estrogen, compared with postmenopausal women with the disease, which may explain why scleroderma tends to be more severe in men, a study has found. Researchers also found that male patients with higher levels of…
Targeting a signaling molecule called CCL24 with a new antibody was seen to lessen inflammation and scarring (fibrosis) in a mouse model of systemic sclerosis (SSc), research shows. The study, “Blockade of CCL24 with a monoclonal antibody ameliorates experimental dermal and pulmonary fibrosis,” was published in journal Annals…
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