Author Archives: Marta Figueiredo PhD

The U.S. Food and Drug Administration has granted orphan drug designation to RLF-100 (aviptadil), Relief Therapeutics’ investigational inhaled therapy for sarcoidosis. Such status is given to treatment candidates with the potential to be safe and effective in rare diseases — defined in the U.S. as those affecting fewer…

The use of Acthar Gel (repository corticotropin injection, or RCI) was among medications noted by both European and U.S. panels of experts, identified as a possible treatment of pulmonary sarcoidosis when other therapies fail or are not tolerated. Notably, the therapy is currently approved to treat active sarcoidosis only…

The U.S. Food and Drug Administration (FDA) has approved beginning clinical trials testing XTMAB-16, Xentria’s investigational TNF-alpha inhibitor for the treatment of sarcoidosis. The first, a Phase 1 trial evaluating the therapy in healthy volunteers, is expected to start in July at the Parexel Baltimore Early Phase…

The Foundation for Sarcoidosis Research (FSR) is holding a two-day virtual patient education summit focused on providing relevant medical content on sarcoidosis, patient and care-partner perspectives, and updates on current and future research in the field. To attend the summit, set for June 12–13, patients and caregivers can…

aTyr Pharma, which is developing a candidate therapy for pulmonary sarcoidosis called ATYR1923, has appointed Andrea Wilson — a sarcoidosis patient and advocate who co-founded the Foundation for Sarcoidosis Research (FSR) — as a patient advisor. “We are pleased during Sarcoidosis Awareness Month to welcome Andrea, a…

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,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to XTMAB-16, Xentria’s investigational TNF-alpha inhibitor for the treatment of sarcoidosis. The designation is given to candidate therapies with the potential to be safe…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Testicular sperm aspiration for in vitro fertilization (IVF) is a successful strategy to overcome infertility associated with methotrexate– and steroid-resistant testicular sarcoidosis, a case study shows. The report documents a case of a man who, within two years of a sarcoidosis diagnosis, showed complete azoospermia (absence…

Severe fatigue affects more than 60% of pulmonary sarcoidosis patients and is associated with worse shortness of breath, daytime sleepiness, anxiety and depression, catastrophizing, functional impairments, and quality of life, according to a Dutch study. The research also showed similar findings for people with idiopathic pulmonary fibrosis (IPF),…