News

Pulmonary arterial hypertension (PAH) represents a major cause of mortality in patients with scleroderma, or systemic sclerosis, accounting for 52% of all deaths — mainly within the first few years after PAH diagnosis, according to recent findings. The fact that PAH is a leading cause of death in scleroderma…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

The presence of B-cells — a type of white blood cell — in the skin of scleroderma patients may predict a more aggressive disease and worsening of skin fibrosis, a study shows. The study, “Characterization of inflammatory cell infiltrate of scleroderma skin: B cells and skin score…

In patients with systemic sclerosis, renal function is generally well-preserved, despite increased stiffness of the arteries that supply the kidneys, a study shows. The study, “Prognostic Factors of Renal Involvement in Systemic Sclerosis,” was published in the journal Kidney & Blood Pressure Research. Systemic sclerosis…

Lenabasum, an investigational treatment of diffuse cutaneous systemic sclerosis by Corbus Pharmaceuticals, was shown to be safe, well-tolerated and of benefit to patients treated for one year in a long-term extension of a Phase 2 trial. Latest results of the JBT101-SSc-001 study (NCT02465437) will be discussed at the European…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

A methotrexate (MTX) treatment protocol that includes the administration of glucocorticoids is superior to phototherapy in treating childhood localized scleroderma, a study found. Nonetheless, researchers suggest MTX should be used preferentially in more severe cases of the disease. The study, “Comparing ultraviolet light A photo(chemo)therapy with…

Early diagnosis and prompt treatment was found to be critical in achieving better long-term outcomes for children with juvenile localized scleroderma, a study reports. The retrospective study, titled “Disease course and long-term outcome of juvenile localized scleroderma: Experience from a single pediatric rheumatology Centre and literature…

Levels of certain important signaling molecules called cytokines are associated with different clinical manifestations in patients with scleroderma, a study suggests. The study, “Different profile of cytokine production in patients with systemic sclerosis and association with clinical manifestations,” was published in the journal Immunology Letters. Cytokines are…