Metabolon Launches M
Metabolon Launches Meta UDx™ Test to Speed Diagnosis of Rare and Undiagnosed Diseases in Children and Adults
28 févr. 2018 10h50 HE | 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...
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APHL Announces Newborn Screening Funding Recipients
19 déc. 2017 10h41 HE | 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 M
Metabolon Launches Meta IMD™ Test to Speed Diagnosis of Inherited Metabolic Disorders
28 sept. 2016 10h05 HE | 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 Rep
EPIRUS Announces Reprioritization of Pipeline to Solely Focus on Biosimilars to Treat Rare Diseases and Key Leadership Changes
09 mai 2016 16h00 HE | 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 Or
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 | 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 B
BioMarin Announces BioMarin RareConnections(TM) Rebranding and Consolidating BioMarin’s Comprehensive Support and Services for Eligible Rare Disease Patients
29 févr. 2016 08h30 HE | 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 2
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 | 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 | 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 &
National Tay-Sachs & Allied Diseases Association Forms Jacob's Cure Fund for Canavan Disease Research
16 oct. 2013 09h55 HE | 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 &
National Tay-Sachs & Allied Diseases Association Receives U.S. Orphan Drug Designation for Novel Gene Therapy
13 juin 2013 06h00 HE | 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...