News

The U. S. Food and Drug Administration (FDA) has granted orphan drug designation to ACE-1334, an investigational therapy that aims to ease scarring in the lungs of people with scleroderma. Acceleron Pharma, the company developing ACE-1334, plans to launch a Phase 1b/2 clinical trial next year,…

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…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Cantargia‘s antibody CAN10, intended to treat people with scleroderma and myocarditis, will be produced by BioInvent under the terms of a new manufacturing agreement, the two companies announced. The investigational therapy is currently in preclinical studies. Cantargia hopes to launch a Phase 1 clinical trial in early 2022,…

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…

Proteins involved in scarring and the escape of immune cells from blood vessels towards nearby tissues are found at high levels in people with diffuse cutaneous systemic sclerosis (dcSSc), a new study reports. The findings also revealed that greater levels of specific proteins correlated with more severe thickening of…

The molecule lysyl oxidase (LOX) plays key roles in promoting skin and lung scarring in scleroderma and may serve as a potential biomarker and therapeutic target for the disease, a study suggests.  The study, “Lysyl Oxidase…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Updated results from the RESOLVE-1 Phase 3 clinical trial show that treatment with lenabasum reduces lung function decline in adults with diffuse cutaneous scleroderma (dcSSc) who are on long-term immunosuppressant therapy. “We are encouraged by the post-hoc [subsequent] analyses pointing to lenabasum’s therapeutic potential,” Yuval Cohen, PhD, CEO of…

Long-term treatment with Ofev (nintedanib) safely and effectively slows lung function decline in adults with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD), according to an early analysis of the SENSCIS-ON extension study. Notably, patients treated with Ofev in both the Phase 3 SENSCIS trial (NCT02597933)…