News

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Pulnovo Medical recently raised an undisclosed amount totaling millions of dollars in investment funding to support clinical trials and product development related to pulmonary arterial hypertension (PAH). “The road of global medical innovation has never been an easy journey,” Cynthia Chen, Pulnovo’s executive president, said in a…

The U.S. Food and Drug Administration (FDA) has approved an intravenous form of Uptravi (selexipag) — one infused directly into the bloodstream — for people with pulmonary arterial hypertension (PAH) temporarily unable to take oral medicines. The new intravenous, or IV, formulation is intended to prevent treatment interruptions in adults…

PulmoSIM Therapeutics has joined with scientists at National Jewish Health hospital and Brown University to advance PT001, its investigational and potentially disease-modifying therapy for pulmonary arterial hypertension (PAH), into clinical trials. The partnership follows PT001 being designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

A protein called TIFA is elevated in the blood cells of people with pulmonary arterial hypertension (PAH), suggesting this protein is involved in the biological processes that drive the disease, according to a new study. The findings were published in the journal Scientific Reports, in the study, “…

An investigational therapy called R-107 completely stopped the progression of pulmonary arterial hypertension (PAH) in a rat model and may become the first medicine to reverse the increasing blood pressure characteristic of the disease, a study suggested. Now, R-107’s developer, Claritas Pharmaceuticals, plans to begin clinical testing…

In people experiencing a pulmonary embolism, higher-than-normal arterial stiffness at the time of hospital discharge increased the risk of pulmonary arterial hypertension (PAH) when evaluated one month later, according to a study in Italy. Given that this patient population is at risk of chronic thromboembolic pulmonary hypertension…

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

An upcoming series of educational virtual events for people with pulmonary arterial hypertension (PAH) and their caregivers will offer insights and perspectives about how to manage life with PAH. The National Broadcast Series will include three talks from August to October. Each of the one-hour talks is scheduled to…

TPN171H, an experimental oral therapy for pulmonary arterial hypertension (PAH), was generally safe and showed a favorable pharmacological profile in healthy volunteers, according to data from a Phase 1 clinical trial. The therapy — previously reported to be a more potent and selective PDE5 suppressor than sildenafil in…

A protein known as cell division cycle protein 2 (CDC2) enhances the proliferation of pulmonary artery smooth muscle cells — those lining the walls of lung arteries — in people with pulmonary arterial hypertension (PAH), a study reported. Additionally, the increased level of CDC2 in these cells was found…