News

FDA Grants Orphan Drug Status to Inhaled Imatinib Formulation

AER-901, an inhaled formulation of imatinib being developed as a treatment for pulmonary arterial hypertension (PAH), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The investigational therapy was designed with the goal of reducing the side effects reported among PAH patients using an…

Home-based Exercise Can Be Viable Alternative in PAH

A six-month at-home rehabilitation program can help improve respiratory muscle strength and physical endurance, while promoting better quality of life, in people with pulmonary arterial hypertension (PAH) That finding from a small clinical trial revealed benefits extending to six months after the end of the program, and suggests this…

Routine, Non-Invasive Tests May Predict PH Severity, Mortality

Routine blood screenings and non-invasive clinical tests may predict disease severity and mortality in people with pulmonary hypertension (PH), a study suggested. Specifically, the red blood cell distribution width (RDW) test, used in hematology laboratories to classify anemia, may help in assessing disease severity, while electrocardiographic ratios may predict…

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Uptravi IV Offers Important Option for Patients, Exec Says

In late July, the U.S. Food and Drug Administration (FDA) approved a new formulation of Janssen‘s Uptravi (selexipag) for use by people with pulmonary arterial hypertension (PAH). Uptravi already was available as an oral tablet. The new formulation is intravenous (IV) — meaning it is administered by an…

Peptide May Prevent PAH, Rat Study Finds

A peptide — a short chain of amino acids, the building blocks of proteins — called BPC 157 may prevent or counteract pulmonary arterial hypertension (PAH) and its complications, according to a preclinical study in a rat model of the disease. The findings suggest that BPC 157 may…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Chemokine Levels in Blood May Mark Risk, Likely Course of IPAH

Certain chemokines — signaling molecules that attract white blood cells to sites of infection — may be blood markers of both a person’s risk of idiopathic pulmonary arterial hypertension (PAH) and this disease’s likely course in a patient, a study found. PAH is considered idiopathic when its cause is unknown…