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Genetic Disorder (misc) News Articles

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Top Breaking News
bluebird bio (BLUE) Touts Impressive Data From Late-Stage Gene Therapy Study     6/26/2017
Shire (SHPG)'s $6.5 Billion Dyax (DYAX) Bet Pays Off as Lanadelumab Wows in Phase III Study     5/18/2017
Safety Concerns Raised as Ionis Pharma (IONS)'s Inotersen Meets Both Primary Endpoints in Late-Stage Study     5/16/2017
Wowsers! FDA Approves BioMarin (BMRN)'s Brineura, the First Drug for Batten Disease     5/2/2017
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

News from Around the Web
CRISPR Gene Editing Can Cause Hundreds Of Unintended Mutations, Columbia University Medical Center Study Reveals     5/30/2017
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

Press Releases
Kamada (KMDA) Submits Proposed Phase III Protocol To FDA For Inhaled Alpha-1 Antitrypsin For Treatment Of Alpha-1 Antitrypsin Deficiency Disease     7/20/2017
Bio Products Laboratory Presents New Phase III Data Demonstrating Prophylactic Coagadex (Coagulation Factor X, Human) Dosing Prevents Or Reduces Bleeding Episodes In Children With Moderate To Severe Hereditary Factor X Deficiency     7/12/2017
Reata Pharmaceuticals, Inc. Receives Orphan Drug Designation For Bardoxolone Methyl For The Treatment Of Alport Syndrome     7/10/2017
Soleno Therapeutics Announces Successful Completion Of FDA Meeting For DCCR In Prader-Willi Syndrome     7/5/2017
Abeona Therapeutics Receives FDA Orphan Drug Designation For ABO-201 Juvenile Batten Disease Gene Therapy Program     6/29/2017
Amicus (FOLD) Submits Japanese New Drug Application For Migalastat For Fabry Disease     6/28/2017
Rhythm And Camurus Announce Positive Initial Data For Extended-Release Delivery Of Setmelanotide For The Treatment Of Rare Genetic Disorders Of Obesity     6/27/2017
NeuroVive (NVP) And Yungjin Pharmaceutical Company Ltd. Start Clinical Development In Genetic Mitochondrial Disease     6/27/2017
Ablynx (ABLYF) Initiates A Single And Multiple Dose Phase I Study Of Caplacizumab In Healthy Japanese Subjects     6/26/2017
bluebird bio (BLUE) Announces Early Data From Phase III Northstar-2 (HGB-207) Study Of LentiGlobin Drug Product At European Hematology Association Annual Meeting     6/23/2017
Kamada (KMDA) WIthdraws MAA For Inhaled Alpha-1 Antitrypsin     6/22/2017
PTC Therapeutics (PTCT) Release: New Analyses of Translarna (Ataluren) Data From ACT DMD Presented At The 12th Annual European Pediatric Neurology Society Congress     6/22/2017
Therachon Receives EMA And FDA Orphan Drug Designation For TA-46 For Achondroplasia     6/20/2017
AGTC (AGTC) Release: IND-Enabling Study Data Published In Human Gene Therapy Clinical Development Support Clinical Development Of AGTC-402 For The Treatment Of CNGA3-Deficient Achromatopsia     6/15/2017
Tarix Orphan Granted Orphan Drug Status By The EMA For TXA127 As Potential Treatment For Epidermolysis Bullosa (EB)     6/12/2017

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