AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2
24 mars 2020 02h13 HE
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Novartis International AG
SMA Type 2 patients between two and five years of age who received Dose B met the primary efficacy endpoint with a remarkable mean increase of 6.0 points in HFMSE scores from baseline to month 12,...
Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)
19 mars 2020 02h15 HE
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Novartis International AG
Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at...
AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome
18 mars 2020 09h27 HE
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C. and CHAPEL HILL, N.C., March 18, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy...
APHL Announces Newborn Screening Systems Quality Improvement Projects Second Cohort Award Recipients
25 févr. 2020 11h15 HE
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Association of Public Health Labs
Silver Spring, MD, Feb. 25, 2020 (GLOBE NEWSWIRE) -- The Association of Public Health Laboratories (APHL) is pleased to announce this year’s recipients of the Newborn Screening Systems Quality...
APHL Announces Newborn Screening Award Recipients
11 févr. 2020 12h01 HE
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Association of Public Health Labs
Silver Spring, MD, Feb. 11, 2020 (GLOBE NEWSWIRE) -- The Association of Public Health Laboratories (APHL) is pleased to announce the fall 2019 recipients of the Expansion of Spinal Muscular Atrophy...
AskBio Launches AskFirst™ Patient Advocacy Program to Educate and Support Participants in Gene Therapy Clinical Trials
29 janv. 2020 10h27 HE
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C., Jan. 29, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, today introduced...
Visiongain launches new report on Gene Therapy R&D and Revenue Forecasts 2018-2028
05 sept. 2018 12h33 HE
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Visiongain Ltd
LONDON, Sept. 05, 2018 (GLOBE NEWSWIRE) -- Gene Therapy Market set to Grow at a CAGR of 41.1% in the first half of the forecast period. Visiongain has launched a new pharma report: Gene Therapy...
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...