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Top Breaking News
Cambridge Biotech Agios (AGIO) Terminates AG-519 for PK Deficiency, But Continues AG-348 For Same Condition     12/19/2016
The Ripple Effect of Alnylam (ALNY)'s Blow-up Yesterday     10/10/2016
Alnylam (ALNY) Stock Craters After Deaths Cause Clinical Trial to End     10/7/2016
Investors Sigh With Relief as bluebird bio (BLUE) Improves Its Gene Therapy Process     9/9/2016
23andMe Ancestry Tests Allegedly Have Issues With People of Color     8/30/2016
Spark Therapeutics (ONCE) Touts Long-Term Positive Phase III Gene Therapy Data     8/11/2016
Vtesse Bags Another $17 Million to Support Further Product Development and Expand Ongoing Clinical Trials     7/25/2016
First-Ever CRISPR Trial Points to Looming Problems     6/30/2016
Marinus Pharma (MRNS)'s Primary Endpoint Not Met in Ganaxolone Phase II     6/28/2016
Chiasma Chief Says Firm May Challenge FDA Over Rare-Disease Drug Rejection     5/12/2016
Astex Pharma (ASTX) and Genentech (RHHBY) Forge a Clinical Trial Deal for Acute Myeloid Leukemia     4/20/2016
FDA Slaps Clinical Hold on Ionis Pharma (IONS) Study While GlaxoSmithKline (GSK) Finalizes Protocol     4/7/2016
Alnylam (ALNY)’s John Maraganore Provides Leadership for RNAi Development and Biotech Industry     4/4/2016
Investors are Irked as BioMarin (BMRN)'s Pegvaliase Shows Mixed Results in Pivotal Study     3/21/2016
Positive Early Stage Pompe Data Allows Sanofi Genzyme (SNY) to Begin Phase III Trial this Summer     3/4/2016

News from Around the Web
Feeling Heavy, Light, Or About Right? Your Genes May Be To Blame, University of Colorado Study     9/1/2016
Should You Get Paid For Your DNA?     3/14/2016
Genetic Testing Company Opens Up Trove Of Cancer Patient Data To Public, Ambry Genetics Reveals     3/10/2016
The Environmental Influence On Epigenetics, National Institute of Environmental Health Sciences Study     7/24/2015
These Superhumans Are Real and Their DNA Could Be Worth Billions     7/22/2015
Project Pinpoints 12 New Genetic Causes Of Developmental Disorders, Wellcome Trust Sanger Institute Study     12/29/2014
Three-Parent Babies May Be Ready In Two Years, Human Fertilization and Embryology Authority Reveals     6/4/2014
Genetic Defect Predicts Cardiac Risk, University of Rochester Study     5/12/2011
Early Work Indicates Drug Used to Treat Alcoholism May Help Those With Fragile X and Autism, Indiana University School of Medicine Study     4/5/2011
Specific Genetic Mutations Associated with Preeclampsia, Hospital for Special Surgery Study     3/23/2011
Mouse Study Suggests New Clues to Celiac Disease, University of Chicago Study     2/10/2011
Blood-Clotting Agent Can Diagnose Fatal Genetic Diseases, University of Manchester Study Finds     2/4/2011
Study Shows Promise for New Drug to Treat Fragile X, Rush University Medical Center Study     1/10/2011
Decoding Fetal DNA From Mother's Blood Opens Door, Study Published in Science Translational Medicine     12/9/2010
Possible New Drug Targets for the Genetic Disorder Noonan Syndrome, Harvard Medical School Study     11/2/2010

Press Releases
NYU Langone Medical Center Release: Company Roots Of Related Genetic Diseases Found In Cell Powerhouses     1/19/2017
aTyr Pharma Release: FDA Grants Fast Track Designation For Company's Resolaris To Treat Limb Girdle Muscular Dystrophy 2B And Removes Partial Clinical Hold For Resolaris     1/18/2017
Tarix Orphan Release: Company Receives Rare Pediatric Disease Designation For TXA127 For Recessive Dystrophic Epidermolysis Bullosa (RDEB)     1/18/2017
MaxCyte, Inc. Release: Company, NIH NIAID Study Published In Science Translational Medicine Demonstrates CRISPR-Cas9 Repair Of X-Linked Chronic Granulomatous Disease (CGD) Gene     1/12/2017
Gemphire Provides Clinical Update     1/5/2017
Fibrocell (FCSC) Announces FDA Fast Track Designation Of FCX-007 For Treatment Of Recessive Dystrophic Epidermolysis Bullosa     1/5/2017
PTC Therapeutics (PTCT) Announces Initiation Of FIREFISH Study In Infant (Type I) SMA Patients     1/5/2017
Sobi Canada Release: Health Canada Approves Orfadin Capsules For Treatment Of Hereditary Tyrosinaemia Type-1 (HT-1)     1/4/2017
Marinus Pharma (MRNS) Receives FDA Orphan Drug Designation For Ganaxolone To Treat Fragile X Syndrome     1/3/2017
Regeneron Genetics Center And Geisinger Health System Study Finds Life-Threatening Genetic Disorder Is Substantially Underdiagnosed     12/22/2016
After Failed Phase III, Auven to Terminate the KIACTA Program     12/21/2016
Novelion (AEGR)’ Subsidiary Launches JUXTAPID (lomitapide) in Japan     12/16/2016
bluebird bio (BLUE) Announces First Patient Treated With LentiGlobin Drug Product In Northstar-2 (HGB-207) Phase III Trial Of Patients With Transfusion-Dependent ?-Thalassemia     12/14/2016
Memorial Hermann Mischer Neuroscience Institute at the Texas Medical Center Release: A Groundbreaking Discovery: Researchers Identify Gene Mutation Linked To Deadly Brain Aneurysms     12/13/2016
Pairnomix Presents Systematic Approach To Identifying Approved Drugs That May Be Repurposed For Treatment Of Rare Genetic Diseases     12/5/2016

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