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Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, people can establish supportive relationships, share experiences and information, and sometimes simply vent. Bionews Services, a leading online health, science, and research publication company, has been rolling out…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

A combination therapy of endothelin receptor antagonists (ERA) plus phosphodiesterase-5 inhibitors (PDE5i) — two different agents that induce blood vessels’ widening and relaxation —improves exercise capacity, and reduces the risk of death and disease worsening in people with pulmonary arterial hypertension (PAH), a review study finds. The…

A Phase 2 clinical trial for levosimendan, a potential treatment for pulmonary hypertension (PH) and heart failure with preserved ejection fraction (PH-HFpEF), will soon have three more active sites for participant enrollment, Tenax Therapeutics announced. There now are 12 clinical sites that have initiated the study, and…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

People who were born prematurely, especially in recent years, are more likely to develop pulmonary arterial hypertension (PAH) later in life, a study says. The findings of the study, “Increased risk of pulmonary hypertension following premature birth,” were published in the journal BMC Pediatrics. PAH is a rare,…

Riociguat increases the risk of death and serious adverse events in people with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP), and should not be prescribed to those patients, final results of a Phase 2 study show. The study, “Riociguat for idiopathic interstitial pneumonia-associated pulmonary hypertension…