Metabolon Launches Meta UDx™ Test to Speed Diagnosis of Rare and Undiagnosed Diseases in Children and Adults
28 févr. 2018 10h50 HE
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Metabolon, Inc.
Meta UDx provides in-depth metabolic information and new insights for difficult-to-diagnose patient cases Expanding LDT clinical testing portfolio demonstrates value of clinical metabolomics for...
APHL Announces Newborn Screening Funding Recipients
19 déc. 2017 10h41 HE
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Association of Public Health Labs
Silver Spring, MD, Dec. 19, 2017 (GLOBE NEWSWIRE) -- For Immediate Release The Association of Public Health Laboratories (APHL) is pleased to announce the recipients of the second round of New...
Metabolon Launches Meta IMD™ Test to Speed Diagnosis of Inherited Metabolic Disorders
28 sept. 2016 10h05 HE
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Metabolon, Inc.
RESEARCH TRIANGLE PARK, N.C., Sept. 28, 2016 (GLOBE NEWSWIRE) -- Metabolon, Inc., the global leader in metabolomics, announced today that Meta IMD™ is now available to physicians as a CLIA...
EPIRUS Announces Reprioritization of Pipeline to Solely Focus on Biosimilars to Treat Rare Diseases and Key Leadership Changes
09 mai 2016 16h00 HE
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EPIRUS Biopharmaceuticals, Inc.
New strategy to focus exclusively on BOW080 (eculizumab biosimilar) and BOW070 (tocilizumab biosimilar) in rare diseases with a combined target market opportunity of close to $6 billion of innovator...
BioMarin Receives Orphan Drug Designation From FDA for First AAV-Factor VIII Gene Therapy, BMN 270, for Patients With Hemophilia A
01 mars 2016 17h42 HE
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BioMarin Pharmaceutical Inc.
SAN RAFAEL, Calif., March 01, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that BMN 270, an investigational gene therapy for the treatment of patients with...
BioMarin Announces BioMarin RareConnections(TM) Rebranding and Consolidating BioMarin’s Comprehensive Support and Services for Eligible Rare Disease Patients
29 févr. 2016 08h30 HE
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BioMarin Pharmaceutical Inc.
SAN RAFAEL, Calif., Feb. 29, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the launch of BioMarin RareConnectionsTM, rebranding its patient-focused offering...
BioMarin Announces 21 Presentations at 12th Annual WORLDSymposium™ 2016 February 29-March 4 in San Diego, California: 6 Oral and 15 Poster
29 févr. 2016 08h25 HE
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BioMarin Pharmaceutical Inc.
SAN RAFAEL, Calif., Feb. 29, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the company will present data in six oral and 15 poster presentations at the...
Actress Patricia Richardson and NORD Join Together to Make a Difference on Rare Disease Day
25 févr. 2014 08h00 HE
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National Organization for Rare Disorders
DANBURY, CT--(Marketwired - Feb 25, 2014) - Millions of Americans will participate in events across the country on February 28 as part of the 6th annual U.S. Rare Disease Day. These activities are...
National Tay-Sachs & Allied Diseases Association Forms Jacob's Cure Fund for Canavan Disease Research
16 oct. 2013 09h55 HE
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National Tay-Sachs & Allied Diseases Association
BOSTON, Oct. 16, 2013 (GLOBE NEWSWIRE) -- National Tay-Sachs & Allied Diseases Association (NTSAD) today announced that it is forming the Jacob's Cure Fund to support research and family services in...
National Tay-Sachs & Allied Diseases Association Receives U.S. Orphan Drug Designation for Novel Gene Therapy
13 juin 2013 06h00 HE
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National Tay-Sachs & Allied Diseases Association
BOSTON, June 13, 2013 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to National Tay-Sachs and Allied Diseases Association (NTSAD) for development...