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Cardiome recently shared an update on the development of its therapeutic portfolio, including Trevyent (treprostinil injection) for the treatment of pulmonary arterial hypertension (PAH). Following the U.S. Food and Drug Administration’s (FDA) refusal last year to approve a new drug application (NDA) submitted by Trevyent’s licensor SteadyMed,…

When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD,  for short. Hunter Pageau, a 12-year-old boy from North Haven, Connecticut, is one of only 80 people in the world known to have SMARD, a motor neuron disease…

Combinations of Reviva Pharmaceuticals’ RP5063 and other pulmonary arterial hypertension treatments improved rats’  lung blood vessel structure and lung function, a study reports. Previous research in rats indicated that RP5063 could improve these measures as a stand-alone therapy. The latest study showed that it worked even better as a combo therapy.

Computer software combined with artificial intelligence can generate advanced imaging techniques for serious diseases such as pulmonary hypertension (PH), improving healthcare, British scientists say. This was the focus of a workshop on digital radiology at Imperial College Academic Health Science Centre (AHSC) that brought together medical researchers, computer scientists, mathematicians…

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…