News

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

News, syndicated

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also ... Read more

February 24, 2021February 24, 2021

Insmed Announces Top-line Results from Phase 1 Trial of TPIP

News, PAH News

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 ... Read more

February 23, 2021February 23, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

News, syndicated

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 ... Read more

February 22, 2021February 22, 2021

FDA Approves Alembic’s Generic Injection of Remodulin for PAH

News, PAH News

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 ... Read more

February 19, 2021February 19, 2021

Remunity Pump on Market for Dispensing Remodulin

News, PAH News

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 ... Read more

February 18, 2021February 18, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

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, ... Read more

February 17, 2021February 17, 2021

More Barriers, Lower Socioeconomic Status Tied to Less Exercise Therapy

News

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 ... Read more

February 15, 2021February 15, 2021

Altered Fat Metabolism May Contribute to PAH Development

News, PAH News, PH News

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 ... Read more

February 12, 2021February 12, 2021

Uptravi Added to Lower Cost Treatment List for PAH Patients in Australia

News, PAH News

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 ... Read more

February 10, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

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 ... Read more