Mesenchymal stem cells (MSCs) derived from umbilical cord blood (UBC) showed more therapeutic benefits than those derived from adipose (fatty) tissue or bone marrow in a rat model of pulmonary arterial hypertension (PAH), a study reports. Specifically, into-the-vein (intravenous, IV) injections…
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While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
PhaseBio Pharmaceuticals‘s investigational therapy pemziviptadil (PB1046) showed a good safety profile and clinically meaningful effects on physical function in a small clinical trial of three people with pulmonary arterial hypertension (PAH). The findings support further evaluation of pemziviptadil as…
Switching from the approved Tyvaso (inhaled treprostinil) nebulizer to Tyvaso DPI — an investigational dry-powder formulation — is safe, effective, and likely more convenient for people with pulmonary arterial hypertension (PAH), top-line data from the Phase 1 BREEZE trial show. These positive findings also were supported by data from…
“Patient journeys” is an initiative designed to help patients and clinicians improve care for rare lung diseases, including pulmonary hypertension (PH). The project, announced in a press release from the European Reference Network on Rare Respiratory Diseases (ERN-LUNG), presents testimonies showing the clinical history and needs of patients through…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
A total of approximately 5.8 new cases per one million inhabitants was found to be the most accurate value for the worldwide incidence of pulmonary arterial hypertension (PAH), according to a review study. That incidence value — the rate of occurrence of new cases of a disease — ranged…
Soluble ST2 (sST2) protein could be an additional non-invasive biomarker for monitoring patients with chronic thromboembolic pulmonary hypertension (CTEPH) undergoing interventional treatment with balloon pulmonary angioplasty (BPA), a study suggests. The study, “Soluble ST2 as a Biomarker for Early Complications in Patients with Chronic Thromboembolic Pulmonary Hypertension Treated…
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…
United Therapeutics is now among the leading sponsors of the Pulmonary Fibrosis Foundation (PFF) Registry, a research data resource for pulmonary fibrosis (PF) that is expanding to include pulmonary hypertension (PH) associated with PF, the foundation announced. Pulmonary hypertension that develops during the course of PF (characterized by scarring…
Tenax Therapeutics has acquired PH Precision Med (PHPM), a private biotech focused on developing imatinib as a treatment for pulmonary arterial hypertension (PAH). “The acquisition of PHPM immediately expands Tenax’s pipeline to include a second de-risked Phase 3 ready candidate with the potential to be the first…
Nebulizer treatment with Tyvaso (inhaled treprostinil) can improve exercise capacity in people with pulmonary hypertension associated with interstitial lung disease (PH-ILD), a study reports. The study, “Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease,” was published in The New England Journal of Medicine. ILD…
