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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…

Patients with scleroderma who undergo lung transplants have similar survival rates to patients who get lung transplants due to other diseases, according to a new study. Freedom from chronic lung allograft dysfunction — a set of pathological conditions that cause a transplanted lung to stop breathing — is also similar…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Rituximab (brand name Rituxan, among others) may be effective in treating patients with diffuse systemic sclerosis, according to a French retrospective study and literature review. Although there are several small-scale reports supporting rituximab as a treatment for systemic sclerosis (SSc) patients, no large-scale studies have been conducted. Researchers found it…

The presence of a specific scleroderma subtype and autoantibodies may be reliable factors to identify a distinct cancer risk in patients with scleroderma, according to a large prospective study. The study, “Autoantibodies and scleroderma phenotype define subgroups at high-risk and low-risk for cancer,” was published in…