News

Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…

Higher blood levels of hepatoma-derived growth factor (HDGF), involved in blood vessel remodeling, associated with greater disease severity and mortality risk in pulmonary arterial hypertension (PAH) patients in a large biobank study. Based on additional work in a rat model, its researchers suggested these outcomes were due to the factor’s…

Newly revealed biological mechanisms that underlie the development of pulmonary arterial hypertension (PAH) support activating BMPR2, the protein mutated in most familial PAH cases, as a treatment strategy, according to a study that investigated human cells and mouse models. A recent case series showed potential for the BMPR2-activator…

Supplemental long-term oxygen therapy is the only effective treatment for people with pulmonary hypertension (PH) associated with chronic obstructive pulmonary disease (COPD) who have low blood oxygen, according to a systematic review of studies. Such long-term oxygen treatment “may mildly reduce severity of PH, slow PH progression over time,…

The short-term survival rate of patients with both human immunodeficiency virus (HIV) and pulmonary arterial hypertension (PAH) — dubbed HIV-PAH — is similar to that of people with idiopathic or familial forms of PAH, according to the results of a Spanish study spanning 20 years. Patients with HIV-PAH also…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Maternal and fetal complications are frequent across pulmonary hypertension (PH) groups, with about a third of patients experiencing an uneventful pregnancy, a small single-center study shows. While PH due to left heart disease is often thought to carry a lower risk of pregnancy complications than pulmonary arterial…

Pulmonary hypertension (PH) has a profound impact on the identity and sexuality of the women who live with the disease, a new study reports. The study, “Sexual and Reproductive Health in Women with Pulmonary Hypertension: A Qualitative Study,” was published in Archives of Sexual Behavior. The impact…

Greater stiffness in the pulmonary arteries, those supplying blood from the heart to the lungs, and problems with the workings of the heart’s right ventricle were evident in obese adults with no known cardiovascular disorder, indicating a possible risk of pulmonary hypertension (PH), according to a recent study. “This study…

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…

Depression and anxiety are common in people with pulmonary hypertension (PH), especially for those living in Asia, a review of 24 studies involving 2,161 participants has found. Researchers noted that “increased awareness amongst physicians regarding the vulnerability of PH patients to psychiatric conditions is key,” and that the “findings…

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.,…