News

A stem cell transplant can lessen skin thickness and improve lung function in people with scleroderma, a longitudinal study found. These findings are in agreement with previous reports, its researchers state, and they support the use of stem cell transplants as a treatment option for patients with severe and…

An elderly woman with scleroderma limited to her skin also developed the autoimmune disorder ANCA‐associated vasculitis (AAV), which led to nerve damage in her lower limbs, a case study described.  The researchers recommended that patients with scleroderma who experience tingling…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Although not a distinctive symptom of scleroderma, lower back pain is a major contributor to the chronic pain felt by people in the earliest disease stages, a study based on a patient viewpoints reported. Physicians should pay more attention to lower back pain and other non-specific symptoms of scleroderma,…

High doses of corticosteroids may be given alongside rituximab, used at lower doses, without affecting the kidneys of people with scleroderma, a study reports. The retrospective study, “Effect of short interval rituximab and high‐dose corticosteroids on kidney function in systemic sclerosis: long‐term experience of…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Patients with scleroderma have poor core stability, as measured by trunk muscle strength and endurance, affecting both their physical capacity and daily performance, a Turkish study found. Previous studies largely neglect trunk muscles in scleroderma patients, focusing instead on upper and…

Common barriers to physical activity in people with scleroderma range from fatigue and poor hand dexterity to symptoms like gastrointestinal discomfort and stiff joints, a large and international patient survey reports. Adjustments made to promote activity, like controlled and slow-movement exercises, regular stretching, and protective and warm clothing, however, were…

The U.S. Food and Drug Administration (FDA) has accepted and granted priority review to an application for Esbriet (pirfenidone) as a treatment for unclassifiable interstitial lung disease (UILD). The supplemental new drug application was submitted by Genentech, Esbriet’s developer and a member of the Roche Group, based on…