The Best Thanksgiving
26 nov. 2019 17h59 HE
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The Children's Inn at NIH
Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) -- One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow...
Syneos Health Releases Report Identifying Barriers to Payer Use of RWE in Rare Disease and Recommendations for Advancing Payer Engagement
21 nov. 2019 09h02 HE
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Syneos Health, Inc.
MORRISVILLE, N.C. and NEW YORK , Nov. 21, 2019 (GLOBE NEWSWIRE) -- Syneos Health® (Nasdaq:SYNH), the only fully integrated biopharmaceutical solutions organization, today released “Real World Value:...
ProQR Receives Orphan Drug Designation from FDA for QR-1123 for Autosomal Dominant Retinitis Pigmentosa
21 nov. 2019 07h00 HE
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ProQR Therapeutics N.V.
LEIDEN, Netherlands and CAMBRIDGE, Mass., Nov. 21, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of...
CENTOGENE Signs New Collaboration with PTC Therapeutics for Global Diagnostic Testing Program
18 nov. 2019 08h00 HE
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Centogene AG
CAMBRIDGE, Ma. and ROSTOCK, Germany, Nov. 18, 2019 (GLOBE NEWSWIRE) -- Centogene N.V. (NASDAQ: CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and...
Retrophin to Present at the Jefferies 2019 London Healthcare Conference
14 nov. 2019 16h30 HE
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Retrophin, Inc.
SAN DIEGO, Nov. 14, 2019 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ: RTRX) today announced that Eric Dube, Ph.D., chief executive officer, will present at the Jefferies 2019 London Healthcare...
Rubius Therapeutics Reports Third Quarter 2019 Financial Results and Operational Progress
14 nov. 2019 08h00 HE
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Rubius Therapeutics
CAMBRIDGE, Mass., Nov. 14, 2019 (GLOBE NEWSWIRE) -- Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an...
CENTOGENE Signs Data Access and Collaboration Agreement with Pfizer Inc.
13 nov. 2019 03h23 HE
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Centogene AG
CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, Nov. 13, 2019 (GLOBE NEWSWIRE) -- CENTOGENE (Nasdaq: CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical...
Retrophin Reports Third Quarter 2019 Financial Results
30 oct. 2019 16h01 HE
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Retrophin, Inc.
Enrollment on-track in pivotal Phase 3 clinical trials to support registration of sparsentan for patients with FSGS and IgAN Third quarter revenues rose nine percent to $44 million ...
Retrophin to Report Third Quarter 2019 Financial Results
16 oct. 2019 16h30 HE
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Retrophin, Inc.
SAN DIEGO, Oct. 16, 2019 (GLOBE NEWSWIRE) -- Retrophin, Inc. (NASDAQ: RTRX) today announced it will report third quarter 2019 financial results on Wednesday, October 30, 2019 after the close of the...
FDA Approves SCENESSE® For Genetic Disorder
08 oct. 2019 19h11 HE
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Clinuvel Pharmaceuticals Limited
MELBOURNE, Australia, Oct. 08, 2019 (GLOBE NEWSWIRE) -- The US Food and Drug Administration (FDA) has approved a new drug as the first ever treatment for a rare genetic metabolic disorder which...