News

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Levosimendan improved right ventricular function in a rat model of pulmonary hypertension, supporting its potential as an effective therapy for the disease, a study shows. The study, “Levosimendan improves cardiac function and myocardial efficiency in rats with right ventricular failure,” was published in the journal Pulmonary…

MannKind Corporation will start enrollment for a Phase 1 trial to test its treprostinil inhaled therapy for the treatment of pulmonary arterial hypertension (PAH). The trial will be conducted under an Investigational New Drug (IND) application filed with the U.S. Food and Drug Administration (FDA). The company’s…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Canada’s Quebec Province has agreed to provide public health insurance coverage of Actelion‘s pulmonary arterial hypertension therapy Uptravi (selexipag). The pan-Canadian Pharmaceutical Alliance (pCPA), a group that represents Canada’s provinces in trying to keep drug costs in line, played a key role in the price negotiations on Uptravi that…

The healthcare costs of Americans with pulmonary arterial hypertension (PAH) are about the same before and after treatment starts, mainly because high treatment costs offset fewer hospitalizations, a study shows. The research, “Treatment Patterns and Associated Health Care Costs Before and After Treatment Initiation Among Pulmonary Arterial Hypertension…

An Ochsner Health System facility in New Orleans has received Pulmonary Hypertension Association  care center accreditation. The association accredits care centers that can assure patients receive top PH diagnosis and treatment. Ochsner’s PH program is located at the John Ochsner Heart and Vascular Institute. It is one of only two accredited…

Family and friends caring for people with rare diseases face emotional, financial, physical, and social burnout, partly because outside sources of support are limited, the first-of-its-kind U.S. survey shows. Global Genes and the National Alliance for Caregiving released the findings, which Greenwald & Associates conducted. It covered 1,406 family members caring…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

The National Institutes of Health (NIH) awarded a $2.8 million Fast Track Small Business Innovation Research (SBIR) grant to PhaseBio Pharmaceuticals to advance the clinical development of PB1046, the first sustained-release analogue of the natural vasoactive intestinal peptide (VIP), in patients with pulmonary arterial hypertension (PAH). In…

Many people with chronic kidney disease also have pulmonary hypertension, a condition that increases their risk of heart problems and death, a review of 16 studies found. Doctors refer to health problems that can damage the heart muscle as cardiovascular events. They include a heart attack, an irregular heartbeat, or a…

A new cardiac surgical procedure to treat mitral valve disease, which is minimally invasive, does not require open-heart surgery, and offers faster recovery, is being tested in an upcoming clinical trial.  Mitral valve disease occurs when the heart’s mitral valve — which has…