PhaseBio Pharmaceuticals has enrolled and dosed its first patient with pulmonary arterial hypertension (PAH) in a Phase 1 clinical study assessing the safety, tolerability and hemodynamic response to its PAH drug candidate PB1046. Participants are all adult PAH patients with a permanently implanted hemodynamic monitor (CardioMEMS HF System). PB1046,…
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Debbie Drell, director of membership at the National Organization for Rare Disorders (NORD), knows all too well about rare diseases. Her older sister, Alex Flipse, was diagnosed with pulmonary hypertension (PH) in September 1998 after enduring five years of worsening symptoms. Drell was told she also had a rare form…
Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
A compound that scientists have been examining as a cancer therapy reduced blood pressure in pulmonary hypertension patients’ lungs, according to a clinical trial. Researchers tested the therapy, dichloroacetic acid, in 20 patients with pulmonary arterial hypertension, or PAH. Although all responded, some with particular gene mutations failed to respond as well,…
Pulmonary hypertension in patients with sarcoidosis is associated with poor prognosis, despite treatment with a targeted therapy, a new study shows. The study “Management and long-term outcomes of sarcoidosis-associated pulmonary hypertension,” was published in the European Respiratory Journal. Patients with sarcoidosis frequently develop pulmonary hypertension. In…
Male gender and lung function parameters have been identified as risk factors for progression of pulmonary arterial hypertension in patients with systemic sclerosis-PAH (SSc-PAH), a study shows. Part of the larger, multi-center, DETECT study, these results are reported in the research, “Factors associated with disease progression in early-diagnosed pulmonary arterial hypertension…
Blocking the enzyme phosphodiesterase 5 (PDE5) is a potential new therapeutic strategy to treat early pulmonary hypertension (PH) in patients who have had a heart attack, a study suggests. The research, “Pulmonary vasodilation by phosphodiesterase 5-inhibition is enhanced and nitric oxide-independent in early pulmonary hypertension after myocardial infarction,”…
The U.S. Food and Drug Administration (FDA) has approved a new, third-generation inhalation device, the TD-300/A, for use with Tyvaso (treprostinil) Inhalation Solution, United Therapeutics Corporation announced. Tyvaso is a prostacyclin vasodilator indicated for the treatment of pulmonary arterial hypertension (PAH) to improve exercise ability. Tyvaso was…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
A heart chamber malfunction leads to worse survival in pulmonary arterial hypertension (PAH) patients who have had a double lung transplant, a study reports. The malfunction, known as diastolic dysfunction, occurs when the heart’s left ventricle is unable to fill up with blood before pumping it out to the body. Researchers…
Patients with pulmonary arterial hypertension (PAH) taking Arena Pharmaceuticals‘ next-generation therapy Ralinepag (APD811) showed significant improvements in blood flow and exercise capacity in a Phase 2 clinical trial. Results of the study (NCT02279160) will be presented at the American College…
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
