LEXINGTON, Mass. and AMSTERDAM — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, a rare, inherited neurodegenerative disorder...
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LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...
LEXINGTON, Mass. and AMSTERDAM, Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease....
LEXINGTON, Mass. and AMSTERDAM, the Netherlands — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary endpoint,...
Nijmegen, The Netherlands – TB is a major threat to public health, being among the leading causes of death worldwide. In 2021, the disease claimed the lives of 1.6 million people, making it the second leading infectious killer after COVID-19.[1] Drug-resistant TB and long treatment regimens have increased the urgency...
NEW YORK, NY — Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO’s members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD), set to be...
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...
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...