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Top Breaking News
Wowsers! FDA Approves BioMarin (BMRN)'s Brineura, the First Drug for Batten Disease     4/28/2017
FDA Calls Regeneron (REGN)'s Evinacumab a Breakthrough     4/7/2017
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

News from Around the Web
CRISPR Cousin SHERLOCK May Be Able To Track Down Diseases, Broad Institute Scientists Say     4/14/2017
Alnylam (ALNY)'s Patisiran, Chronicle Of A Death Foretold     4/11/2017
This DNA-Testing Company Is Offering Patients A Chance To Help Find A Cure For Their Conditions     4/7/2017
Bangladeshi Girl May Be First Female With 'Tree Man Syndrome'     2/2/2017
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

Press Releases
ProQR Therapeutics B.V. (PRQR) Announces Clearance Of IND Application To Start Clinical Trial For QR-110 In Leber’s Congenital Amaurosis Type 10 Patients     4/27/2017
PhaseRx (PZRX) Receives Positive Opinion For Orphan Drug Designation For PRX-OTC From EMA     4/25/2017
Sucampo (SCMP) Release: New Study Published In PLOS ONE Advances Science Around VTS-270 For Treatment Of Niemann-Pick Disease Type C1     4/18/2017
Protalix (PLX) Announces New Preclinical Results Demonstrating A Positive Effect Of Pegunigalsidase Alfa (PRX-102) On Small-Fiber Neuropathy In Fabry Disease Models Compared To Commercially Available Enzyme Replacement Therapies     4/18/2017
CRISPR Cousin SHERLOCK May Be Able To Track Down Diseases, Broad Institute Scientists Say     4/14/2017
Ovid Announces Initiation Of Phase I Clinical Trial Of OV101 For Adolescents With Angelman Syndrome Or Fragile X Syndrome     4/10/2017
Kadmon Submits Second Abbreviated New Drug Application Filing For Trientine Hydrochloride To FDA     4/4/2017
Invitae Announces New Partnership With Alnylam (ALNY) To Provide TTR Gene Testing For Patients Suspected Of Having Hereditary ATTR Amyloidosis     4/3/2017
Horizon Pharma (HZNP) To Present Data On RAVICTI (Glycerol Phenylbutyrate) Oral Liquid For Urea Cycle Disorder Patients Aged Two Months To Two Years     3/22/2017
Abeona Therapeutics Receives Orphan Drug Designation In The European Union For EB-101 Gene Therapy Clinical Trial For Epidermolysis Bullosa     3/8/2017
90% Of The Cases Analysed Are Carriers Of At Least One Genetic Mutation: iGenomX     3/2/2017
Attune Announces Late-Breaking Poster Presentation Of ATN-249, An Oral Kallikrein Inhibitor For The Treatment Of HAE     3/1/2017
Fibrocell (FCSC) Announces Dosing Of First Patient In Phase I/II Clinical Trial Of FCX-007 Gene Therapy For Treatment Of Recessive Dystrophic Epidermolysis Bullosa     2/23/2017
Abeona Therapeutics Provides Update From ABO-102 Phase I/II MPS IIIA Clinical Trial At The 13th Annual WORLDSymposium 2017     2/17/2017
Invitae Release: New Research Suggests Broader Genetic Testing Of Prostate Cancer Patients May Be Warranted To Identify Risk Of An Inherited Mutation That Might Inform Treatment     2/17/2017

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