Author Archives: Marisa Wexler MS

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Galapagos NV and Gilead Sciences are discontinuing all clinical trials with the investigational medication ziritaxestat. This includes the long-term extension of the Phase 2a NOVESA trial in scleroderma, as well as the Phase 3 ISABELA program in idiopathic pulmonary fibrosis (IPF). The decision is based on…

Small genetic variations that alter gene activity could play a role in the development of scleroderma and open new avenues for treating the disorder, a study indicates. Many of the genes identified through this work also show unusual levels of activity in tissues often affected in this disease. The study,…

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…