Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response to COVID-19 are expected to have...
Latest News
Study demonstrated safety and tolerability of Tyvaso DPI™ in patients with PAH transitioning from Tyvaso® Inhalation Solution A separate study in healthy volunteers demonstrated comparable treprostinil exposure between Tyvaso DPI and Tyvaso Inhalation Solution Tyvaso DPI NDA filing anticipated in April 2021 with priority review SILVER SPRING, Md. and RESEARCH...
SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C. – United Therapeutics Corporation (Nasdaq: UTHR) announced today that the first patient has enrolled in the phase 3 TETON study, which is expected to evaluate approximately 396 adult patients with idiopathic pulmonary fibrosis (IPF). This 52-week study will evaluate the impact of...
SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C. – United Therapeutics Corporation (Nasdaq: UTHR) today announced the submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for Tyvaso DPI™, a novel dry powder inhalation formulation of treprostinil, for the treatment of pulmonary arterial hypertension...
SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C. – United Therapeutics (Nasdaq: UTHR), a public benefit corporation with a purpose to provide a brighter future for patients, today announced that the company has been named on Newsweek’s list of America’s Most Responsible Companies 2022. This award is presented by Newsweek...
Birmingham, England – Societal acceptance of autism varies considerably across different countries, with lowest levels of acceptance found in Japan and Belgium, new research shows. A survey of 306 autistic individuals from eight countries revealed that around three quarters of respondents do not feel accepted, or only sometimes feel accepted,...
Southampton, UK – Scientists have taken a major step towards developing a blood test that could identify millions of people who spread tuberculosis unknowingly. A breakthrough study has discovered a group of biological markers that are found in high levels among infectious patients. The researchers hope the findings will pave...
Toronto, Canada – Researchers at the University of Toronto and Sinai Health have created a new platform to identify proteins that can be co-opted to control the stability of other proteins — a new but largely unrealized approach to the treatment of disease. The researchers developed a method to interrogate the...
Tokyo, Japan – The onset of psychosis can be predicted before it occurs, using a machine-learning tool which can classify MRI brain scans into those who are healthy and those at risk of a psychotic episode. An international consortium including researchers from the University of Tokyo, used the classifier to...
London, UK – Sight loss in certain inherited eye diseases may be caused by gut bacteria, and is potentially treatable by antimicrobials, finds a new study in mice co-led by a UCL and Moorfields researcher. The international study observed that in eyes with sight loss caused by a particular genetic...