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Increased Education Crucial to Improving Rare Disease Care, Survey Finds

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…

Activating BMPR2 Protein Supported as PAH Treatment Strategy

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…

Oxygen Therapy Is Only Effective Treatment for COPD With PH: Study

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,…

Obesity Can Damage Heart in Ways That Risk Pulmonary Hypertension

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…

#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…