Marisa Wexler, MS,  senior science writer—

Scleroderma treatment TMB-003, which aims to improve patients’ quality of life by reducing the scarring that characterizes the autoimmune disorder, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). This designation is awarded to investigational therapies intended to improve treatment for rare diseases,…

A new documentary, “Beyond Breathless,” will showcase the journey of those affected by interstitial lung disease (ILD) through the real experiences of people living with rare lung diseases, their loved ones, and healthcare professionals. The documentary, by pharmaceutical company Boehringer Ingelheim, will premier at noon EST Saturday (Dec. 5) on…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Autologous hematopoietic stem cell transplant (AHSCT) — replacing patients’ damaged bone marrow with their own healthy blood stem cells —  improves health-related quality of life among people with scleroderma to a greater extent than conventional treatment, a study shows. The study, “Autologous hematopoietic stem cell…

Talaris Therapeutics has raised $115 million in financing, which will be used to support the clinical development of its lead therapeutic candidate, FCR001, designed to promote immune tolerance and allow patients to stop taking immunosuppressive medications after a transplant. The newly raised funds…

Following the onset of the COVID-19 pandemic, people with scleroderma experienced substantial increases in anxiety, but not depression, a new study has found. Results also show that older patients and those with greater financial resources experienced smaller increases in anxiety. The study, “Changes in mental health…

A protein known as TL1A drives lung fibrosis, or scarring, in scleroderma, a new study suggests. The findings suggest that blocking TL1A could be an effective therapeutic strategy in scleroderma and other conditions characterized by lung fibrosis, including severe asthma, and idiopathic pulmonary fibrosis. Titled, “…

Detailed characteristics of 12 cases of lung cancer in people with scleroderma were described in a new study. Notably, all patients were women and most had interstitial lung disease (ILD). The study, “Clinicopathological characteristics of lung cancer in patients with systemic sclerosis,” was published…

Obstructive sleep apnea (OSA) is associated with an increased risk of pulmonary hypertension in people with scleroderma, a study suggests. The study, “Impact of concomitant obstructive sleep apnea on pulmonary involvement and main pulmonary artery diameter in adults with scleroderma,” was published in the journal Sleep…