News | News By Subject | News by Disease News By Date | Search News

Genetic Disorder (misc) News Articles

Get Our FREE
Industry eNewsletter
Top Breaking News
Alnylam (ALNY) Stock Catapults as Genetic Disease Drug Meets All Phase III Goals     9/20/2017
Amicus (FOLD) Dumps Lead Rare Disease Drug After Phase III Trial Failure     9/14/2017
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

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
Arbutus Biopharma (ABUS)’ LNP Licensee Alnylam (ALNY) Announces Positive Phase III Results For LNP-Enabled Patisiran Program     9/21/2017
Akari to Push Lead Drug Into Phase III For PNH In Q1 2018, Stock Surges     9/21/2017
Amicus (FOLD) Release: FDA Grants Orphan Drug Designation For ATB200/AT2221 For Pompe Disease     9/21/2017
ProQR Therapeutics (PRQR) Presents In Vivo Proof Of Concept Data For The Axiomer RNA Editing Platform Technology     9/21/2017
MyoKardia Presents Additional Positive Data From Phase II PIONEER-HCM Study Of Mavacamten (Formerly MYK-461) At The Heart Failure Society Of America’s 21st Annual Scientific Meeting     9/19/2017
Soleno Therapeutics Presents Updated Safety And Efficacy Data From Clinical Trial Of DCCR For Treatment Of Prader-Willi Syndrome     9/15/2017
Amicus (FOLD) Announces Approval For Galafold (Migalastat) For Treatment Of Fabry Disease In Canada     9/15/2017
Akcea Therapeutics And Ionis Pharma (IONS) Announce Filing Of New Drug Submission For Volanesorsen To Health Canada     9/13/2017
bluebird bio (BLUE) Announces First Patient Treated In Expansion Cohort Of CRB-401, Phase I Study Of Anti-BCMA CAR T Therapy Bb2121     9/13/2017
Editas Medicine (EDIT) Initiates Clinical Natural History Study To Evaluate Patients With Leber Congenital Amaurosis Type 10 (LCA10)     9/12/2017
Aeglea (AGLE) Doses Two Patients In Repeat Dose Part Of Phase I/II Clinical Trial For The Treatment Of Arginase 1 Deficiency     9/6/2017
Crinetics Awarded Two SBIR Grants To Develop New Therapeutics For Congenital Hyperinsulinism And Cushing’s Disease     9/6/2017
Biocryst (BCRX) Announces Positive Results From Its Apex-1 Phase II Trial In HAE     9/5/2017
Amyloid Publishes Long-Term Data Analysis From Pfizer (PFE) Suggesting Tafamidis Delays Progression Of TTR-FAP, A Rare Disease     8/31/2017
Dimension Therapeutics (DMTX) Commences Patient Dosing In Global, Multi-Center Phase I/II Clinical Trial Of DTX301 In Ornithine Transcarbamylase (OTC) Deficiency     8/31/2017