Intravenous infusions of treprostinil can improve disease severity in children with pulmonary hypertension (PH) associated with congenital diaphragmatic hernia (CDH), a birth defect that occurs when the diaphragm does not form properly, a new study shows. However, these babies have a greater likelihood of needing extracorporeal respiratory support,…
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A Phase 1 clinical trial (NCT03464864) found that treprostinil can be safely delivered at varying doses using Technosphere, a novel inhalation device, MannKind, the device’s developer, announced. Treprostinil — a vasodilator — was approved by the U.S. Food and Drug Administration (FDA) in 2002 for the treatment of pulmonary…
Denovo Biopharma recently entered an exclusive global license agreement with Stanford University School of Medicine to further develop and market its lead compound, DB102 (formerly called enzastaurin), as a potential treatment for pulmonary arterial hypertension (PAH) and emphysema. In research in preclinical models at Stanford, DB102 was found…
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…
A molecular mechanism involving the tight balance between two proteins — Gal-1 and CaV1.2 — was found to regulate the behavior of blood vessel cells, directly contributing to hypertension. This finding provides new insights into the underlying processes that trigger arterial contraction and hypertension, and its researchers suggest that a…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
A new Phase 2 study evaluating Tenax Therapeutic’s investigational levosimendan as a therapy for pulmonary hypertension (PH) associated with heart failure and preserved left ventricular ejection fraction (PH-HFpEF) — a type of PH related to left heart disease – will soon begin recruiting participants, the company announced. Patients…
Chronic thromboembolic pulmonary hypertension (CTEPH) may be an inherited genetic disease, a new study suggests. The results were recently presented at the American Thoracic Society (ATS) 2018 annual conference May 18-23 in San Diego. CTEPH, a rare form of pulmonary hypertension, is caused by blood clots that travel to the…
Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
Direct oral anticoagulants — a type of blood thinner medication — are a safe and effective treatment for chronic thromboembolic pulmonary hypertension (CTEPH), according to a small study. The study, “Use of direct oral anticoagulants for chronic thromboembolic pulmonary hypertension,” was published in the journal Clinics.
Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…
