Top-line data from a Phase 1 clinical trial has found that treprostinil palmitil inhalation powder (TPIP) — a dry powder form of a treprostinil precursor that is being developed by Insmed for treating pulmonary arterial hypertension (PAH) — was safe and well-tolerated in healthy volunteers. Results also showed…
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Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…
The U.S. Food and Drug Administration (FDA) has approved Alembic Pharmaceuticals’ injectable formulation of treprostinil, a generic version of Remodulin, to treat patients with pulmonary arterial hypertension (PAH), the company announced. Like the branded product, this generic version of United Therapeutics’ treprostinil will be available in multiple-dose vials…
Remunity, an innovative pump for Remodulin (treprostinil) to treat adults with pulmonary arterial hypertension (PAH), has launched commercial sales, according to the device’s developer, United Therapeutics. The Remunity system allows constant subcutaneous (under-the-skin) delivery of Remodulin (treprostinil). The therapy mimics the effects of prostacyclin, a natural vasodilator…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
People with pulmonary hypertension are less likely to be referred to exercise therapy if they are of lower socioeconomic status or perceive more barriers to accessing such therapy, a study shows. These findings highlight the need for increased education about the benefits of exercise for people with pulmonary hypertension,…
Adipose (fat) tissue may contribute to the development of pulmonary arterial hypertension (PAH) due to certain metabolic alterations found, for the first time, in patients and also in rodent models of the condition, a study suggests. The study, “…
Australia’s government has taken a step to ensure that Uptravi (selexipag), an oral therapy for pulmonary arterial hypertension (PAH), is available to people affected by the disease. Janssen’s treatment is now included in the country’s Pharmaceutical Benefits Scheme (PBS) listing, meaning it is available to residents in this program…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons and…
Statins have protective effects and can benefit patients with both pulmonary hypertension (PH) and chronic obstructive pulmonary disease (COPD), according to a large retrospective study in Taiwan. The study, “Statins for the Treatment of Pulmonary Hypertension in Patients with Chronic Obstructive Pulmonary Disease,” was published…
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…
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…
