News

#RAREis Representation Program Promotes Equity, Diversity

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

Ferrer Will Market Tyvaso for PH-ILD in More Countries

Ferrer has acquired exclusive rights to commercialize Tyvaso (inhaled treprostinil) for the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD) outside North America, Japan, China, and Israel. The therapy, developed by United Therapeutics, is currently approved for that indication only in the U.S.,…

Rare Disease Day Panel Opens Window to Patient Experience

BioNews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…

In Children, Double Lung Transplant Can Normalize Heart Function

A bilateral or double lung transplant can rescue heart function in children and adolescents with pulmonary hypertension (PH), a U.S. single-center study reports. Specifically, a transplant was found to effectively normalize right ventricular (RV) function of the heart in PH patients up to age 21. Additionally, RV dysfunction prior to…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

Administrative Data Can Identify PH to Aid in Disease Research

Hospital-based administrative data can be used to identify patients with pulmonary hypertension (PH) in medical records, according to a study done in Canada. These results, if validated, would pave the way for advances in the screening and treatment of PH, and help investigators improve epidemiological research into this rare…