The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to efzofitimod (ATYR1923) to treat interstitial lung disease (ILD) in people with systemic sclerosis (SSc). The therapy was developed by aTyr Pharma for treating severe diseases affecting the lungs. Efzofitimod was designated as an orphan drug…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
Country music artist Ashley Barron has been named celebrity ambassador of the National Scleroderma Foundation, a role she will take up to increase understanding and awareness of scleroderma as she takes her music on tour this spring and summer throughout the United States. “I am so honored…
UVA1 phototherapy, a form of treatment that uses light for skin disorders, helps ease skin lesions in people with localized scleroderma by regulating certain genes responsible for inflammation and scarring, a study has found. The study, “Clinical and laboratory characterization of patients with localized scleroderma and response to…
Treatment with stem cells grown into a skin-like structure called an organoid reduced inflammation and fibrosis (scarring) in a mouse model of localized scleroderma, a new study found. The study, “Application of an iPSC-Derived Organoid Model for Localized Scleroderma Therapy,” was published in Advanced Science.
A differential blood cell count — a routine test that tells how many cells of each type there are in the blood — may be able to foretell the course of disease in patients with systemic scleroderma (SSc), according to a U.S. study. Higher numbers in both neutrophil counts…
A combination of ultrasound and therapy that includes joint manipulation and massage improved hand function and ulcer wound healing in a small group of systemic sclerosis (SSc) patients with skin involvement, a study reported. “Therapeutic [ultrasound ] combined with manual therapy should be used as additional intervention to manage…
In patients with systemic sclerosis, low levels in the blood of two proteins, SIRT1 and SIRT3, are associated with poor lung function, widespread skin scarring, and inflammatory damage to the small blood vessels, a recent study shows. Results suggest that SIRT1 and SIRT3 may be useful biomarkers to predict…
Several clinical characteristics in people with systemic sclerosis (SSc) — specifically, being male, older age, diffuse SSc forms, and inflammation — were associated with a greater accumulation of organ damage over time in a recent study. The level of damage accumulation, called a damage trajectory, also correlated with mortality…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
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