A 42-year-old man with scleroderma responded well to a second stem cell transplant after experiencing a disease relapse with lung involvement following his initial transplant, according to a recent report. Improvements in skin thickening and lung function were quickly seen, and the patient was still in remission two years…
In a real-world study, the systemic sclerosis (SSc) medication RoActemra (tocilizumab) failed to significantly lessen skin and lung scarring compared with standard of care treatment. RoActemra, an approved immunosuppressant therapy for systemic sclerosis-associated interstitial lung disease (SSc-ILD), showed no statistically significant differences in efficacy in treating patients in this…
The release of CXCL10 — an inflammatory signaling molecule associated with a worse prognosis in scleroderma — from connective tissue cells and those lining blood vessel walls was significantly weakened with iloprost, a recent study reports. These findings showcase the added benefits of iloprost, commonly used to widen blood vessels…
Being male is linked with a higher risk of mortality among patients with scleroderma, irrespective of the type of autoantibody they carry, according to an analysis from two large databases. The study, “Sex-specific risk of anti-topoisomerase antibodies on mortality and disease severity in systemic sclerosis: 10-year…
The levels of certain metabolites — molecules that take part in metabolism — circulating in the blood may help differentiate pulmonary arterial hypertension (PAH) due to scleroderma (SSc-PAH) from idiopathic PAH, a new study shows. The “metabolic signature” correlated with worse disease severity in PAH tied to SSc.
Between 4% and 6% of people with scleroderma (SSc) were first determined to have an interstitial lung disease (ILD), according to an analysis of a large U.S. insurance claims database. This finding supports evidence of scleroderma’s early impact on lung disease, and “underline the importance of clinical examination and…
A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…
The immunosuppressant rituximab may help stabilize lung function in people with systemic sclerosis-related interstitial lung disease (SSc-ILD), a study suggests. The study,“Rituximab treatment for systemic sclerosis associated interstitial lung disease – A case series of 13 patients,” was published in the Internal Medicine Journal. Rituximab, a medication that…
Daily oral treatment with zibotentan, a blocker of the endothelin protein receptor, was found safe, and possibly may be able to improve kidney function in scleroderma patients with kidney disease, a Phase 2 trial shows. The study, “Zibotentan in systemic sclerosis-associated chronic kidney disease: a phase II randomised…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Gesynta Pharma’s GS-248, its oral therapy for people with systemic sclerosis (scleroderma). Orphan drug status aims to encourage therapies for rare diseases through benefits such as seven years of market exclusivity and exemption from FDA fees. “The…
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