Aeglea BioTherapeutics Announces 1-Year Data for Pegzilarginase in Patients with Arginase 1 Deficiency at the 6th Congress of the European Academy of Neurology
May 26, 2020 08:30 ET
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Aeglea BioTherapeutics, Inc.
Pegzilarginase Showed Durable Clinical Response at 56 Week Analysis All Patients Demonstrated a Marked and Sustained Reduction in Plasma Arginine Favorable Safety Profile, Consistent with...
BrightInsight and CSL Behring Announce Global Digital Health Partnership to Improve Treatment Experience for Patients with Rare Diseases
May 06, 2020 08:30 ET
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BrightInsight; CSL Behring
SAN JOSE, Calif. and KING OF PRUSSIA, Pa., May 06, 2020 (GLOBE NEWSWIRE) -- BrightInsight, Inc., the provider of the leading regulated IoT platform for biopharma and medtech, and CSL Behring, a...
aTyr Pharma to Webcast Conference Call Reporting First Quarter 2020 Financial Results
May 06, 2020 08:03 ET
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aTyr Pharma, Inc.
SAN DIEGO, May 06, 2020 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel immunological...
Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak
April 27, 2020 13:00 ET
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FFF Enterprises.com
Temecula, April 27, 2020 (GLOBE NEWSWIRE) -- FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help...
SCENESSE® to be Prescribed in China
April 23, 2020 02:08 ET
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Clinuvel Pharmaceuticals Limited
MELBOURNE, Australia and HANGZHOU, China, April 23, 2020 (GLOBE NEWSWIRE) -- Australian-based company CLINUVEL is launching SCENESSE® (afamelanotide 16mg) in the People’s Republic of China for the...
CENTOGENE Announces Changes in Supervisory Board
March 17, 2020 15:39 ET
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Centogene AG
CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, March 17, 2020 (GLOBE NEWSWIRE) -- CENTOGENE N.V. (Nasdaq: CNTG), a commercial-stage company focused on rare diseases that transforms real-world...
American Gene Technologies to Present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
March 10, 2020 09:34 ET
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American Gene Technologies
ROCKVILLE, Md., March 10, 2020 (GLOBE NEWSWIRE) -- American Gene Technologies (AGT), a leading gene and cell therapy company in Rockville, Maryland, is pleased to announce its Chief Science Officer C....
Palvella Therapeutics Completes Enrollment in Phase 2/3 Pivotal Study of PTX-022 for Treatment of Pachyonychia Congenita
March 06, 2020 07:00 ET
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Palvella Therapeutics
Wayne, PA , March 06, 2020 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for...
Retrophin to Report Fourth Quarter and Full Year 2019 Financial Results
February 10, 2020 16:30 ET
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Retrophin, Inc.
SAN DIEGO, Feb. 10, 2020 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ: RTRX) today announced it will report fourth quarter and full year 2019 financial results on Monday, February 24, 2020 after the...
Rubius Therapeutics Announces First Patient Dosed in Phase 1b Trial of RTX-134 for the Treatment of Patients with Phenylketonuria
January 30, 2020 08:00 ET
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Rubius Therapeutics
CAMBRIDGE, Mass., Jan. 30, 2020 (GLOBE NEWSWIRE) -- Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an...