Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
The use of the hormone vasopressin successfully stabilized symptoms associated with pulmonary hypertension (PH) in newborns, and avoided the need for more invasive treatment approaches in some infants, according to data from 10 cases. “Although our findings are exploratory and further research is needed to establish safety and efficacy,…
Hyaluronic acid, a major component of the extracellular matrix that supports cell function, directly contributes to the development of pulmonary hypertension (PH) via pulmonary vascular remodeling, a preclinical study has found. By pharmacologically blocking the production of the substance in a PH mouse model, researchers were able to prevent…
Levels of the protein TXNRD1 are reduced in the blood of people with idiopathic pulmonary arterial hypertension (IPAH), suggesting it may be useful as a potential diagnostic marker or treatment target in IPAH, according to a new study. “This study is the first report to prove that TXNRD1 may…
The U.S. Food and Drug Administration (FDA) has approved LungFit PH, Beyond Air‘s device to administer nitric oxide therapy, to treat persistent pulmonary hypertension of the newborn (PPHN). “The FDA approval of LungFit PH enables a new era of nitric oxide therapy and marks a pivotal event for…
The leading causes of death among people with pulmonary hypertension (PH) differ by PH type, a Japanese study has found. When categorized using the World Health Organization (WHO) PH classification, those in group 1 died mostly from PH itself, those in group 3 died more often from…
An under-the-skin infusion of a protein called netrin-1 — originally found to regulate nervous system development — shows promise for lessening pulmonary hypertension (PH), a mouse study suggests. Additionally, the scientists found that three small peptides derived from netrin-1 also are a potential therapy for easing PH. The…
Seven members of the pulmonary hypertension (PH) community — ranging from an 11-year-old advocate diagnosed in infancy to a physician who has led clinical trials, to a longtime fundraiser who’s a retired teacher — have been honored for their dedication and service by the Pulmonary Hypertension Association (PHA).
Pulmonary hypertension (PH) induced in response to exercise may be linked to a higher risk of cardiovascular (CV) disease in patients with human immunodeficiency virus (HIV), a small study found. Those with a higher CV risk had fewer CD4 T-cells, a type of immune cell that drops to extremely…
Treatment with an analog of sotatercept, Acceleron Pharma’s experimental therapy, eased disease-associated inflammation and changes in blood vessel architecture in multiple rodent models of pulmonary arterial hypertension (PAH), a study reported. The study, “Sotatercept analog suppresses inflammation to reverse experimental pulmonary arterial hypertension,” was published in…
A research form of Keros Therapeutics’ investigational therapy KER-012, called RKER-012, reduced lung inflammation and scarring (fibrosis) in a rat model of pulmonary arterial hypertension (PAH). The therapy also prevented an increase in the levels of a heart damage marker, the company announced in a press release. The…
Seralutinib (GB002), an investigational inhaled treatment for pulmonary arterial hypertension (PAH), effectively treated severe disease in two animal models, a study demonstrated. Further, when compared directly, inhaled seralutinib showed greater efficacy than imatinib, an approved cancer therapy also being investigated for PAH. Gossamer Bio, seralutinib’s developer, now is testing…
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