The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
Genetic screening of patients with persistent pulmonary hypertension of the newborn (PPHN) found three rare mutations associated with the condition, suggesting it has a genetic basis, a study reports. The researchers also found that a link between PPHN and genetic variants…
Men with pulmonary arterial hypertension (PAH) are more likely to carry mutations in the BMPR2 gene, a known genetic risk factor for PAH, a study from China found. Male PAH patients had more severe disease and were diagnosed at younger age than women. The results support the therapeutic potential…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
The U.S. Food and Drug Administration (FDA) granted an extra clearance to the Unity Subcutaneous Delivery System for Remodulin (treprostinil) Injection, also known as the Remunity pump, allowing the system to be used with drug cassettes that have been prefilled by specialty pharmacies. The Remunity system allows for constant, subcutaneous…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
You literally can’t miss it in the news these days — millions (if not billions) of people all over the world are talking about the increasing spread of “the new (novel) Coronavirus.” According to the Center for Disease Control (CDC), the earliest cases of COVID-19 began in the city of…
Aria CV announced the end of a funding round that will be used to finance a feasibility study of its medical device, the Pulmonary Hypertension System (Aria CV PH System), in people with pulmonary arterial hypertension (PAH) in the U.S. The series B round, which raised $31 million, was led…
A unique bacterial profile in the guts of patients with pulmonary arterial hypertension (PAH) may contribute to the disease and help predict it, a study found. The identification of specific microorganisms may lead to earlier, less invasive diagnoses, as well as new avenues for treatment. The study,…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. 20 webinar. William Whitman…
The ATS Foundation Research Program and Pulmonary Hypertension Association have announced the 2019 recipients of their partner grants, totaling $210,000, for young researchers dedicated to scientific discovery and better patient care in the field of pulmonary hypertension. An $80,000 Foundation Partner grant was awarded to Rahul Kumar, PhD, a…
Use of Bellerophon Therapeutics’ INOpulse device — which provides nitric oxide to the lungs — significantly improved parameters related to blood flow in the lungs in people with pulmonary hypertension associated with pulmonary fibrosis (PH-PF), top-line data from…
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