41 reports

  • GENETIC DISORDERS MARKET, GLOBAL, SAREPTA ANNUAL REVENUE FORECAST ($BN), 2016-
  • GENETIC DISORDERS MARKET, GLOBAL, CLINICAL TRIAL DURATION BY STAGE OF DEVELOPMENT

Exondys ## binds to RNA and efficiently interferes with gene expression in a sequence-specific manner.

  • Genetic Disorder
  • Market Size
  • Alexion Pharmaceuticals, Inc.
  • Alnylam Pharmaceuticals, Inc.
  • Amgen Inc.

RNA SEQUENCING SERVICES SUCH AS THE RNA QUANTIFICATION SEQUENCING, RNA TRANSCRIPTOME SEQUENCING, LONG NON-CODING RNA SEQUENCING, AND SMALL RNA SEQUENCING; AND CUSTOMIZED SOLUTIONS LIKE THE IMMUNE REPERTOIRE SEQUENCING, SINGLE-CELL SEQUENCING, AND PROTEOMI

  • Genetic Disorder
  • Agilent Technologies, Inc.
  • F. Hoffmann-La Roche Ltd.
  • Illumina, Inc.
  • Natera, Inc.
  • 4.4.3.3 Molecular Target
  • RESPIRATORY DISORDERS THERAPEUTICS MARKET, GLOBAL, PIPELINE BY KEY INDICATIONS AND

DURING CHILDHOOD, ASTHMA IS MORE COMMON IN MALES, WITH A MALE-TO-FEMALE RATIO OF ##:## UP TO PUBERTY, WHEN IT CHANGES TO ##:##.

  • Genetic Disorder
  • Market Size
  • Astellas Pharma Inc.
  • AstraZeneca PLC
  • Biologics, Inc.

The tests discussed under this protocol includes analysis of DNA & RNA as well as chromosomal analysis.

  • Genetic Disorder
  • World
  • Forecast
  • Market Size
  • PerkinElmer, Inc.
  • Cystic Fibrosis Drug Pipeline by Route of Administration

A person inherits two copies of the CFTR gene: one from the mother and one from the father.

  • Cystic Fibrosis
  • Genetic Disorder
  • World
  • AbbVie Inc.
  • Vertex Pharmaceuticals Incorporated

According to WHO, it ranges from ## in ##, ## to ## in ##, ##.

  • Genetic Disorder
  • AbbVie Inc.
  • Allergan plc
  • Pharmaxis Ltd
  • Vertex Pharmaceuticals Incorporated

According to WHO, it ranges from ## in ##, ## to ## in ##, ##.

  • Genetic Disorder
  • AbbVie Inc.
  • Allergan plc
  • Pharmaxis Ltd
  • Vertex Pharmaceuticals Incorporated

You can easily book an appointment with one online.

  • Genetic Disorder
  • AstraZeneca PLC
  • GlaxoSmithKline plc
  • Merck & Co., Inc.
  • Novartis AG

AMINOGLYCOSIDES WORK BY BINDING TO THE BACTERIAL ##S RIBOSOMAL RNA (RRNA) OF ##S RIBOSOMAL SUBUNIT, CAUSING MISREADING OF TRANSFER RNA (T-RNA), LEAVING THE BACTERIUM UNABLE TO SYNTHESIZE THE PROTEINS VITAL TO ITS GROWTH (EHSAN Z AND CLANCY JP, 2015).

  • Genetic Disorder
  • Forest Laboratories, Inc.
  • Novartis AG
  • Pharmaxis Ltd
  • Vertex Pharmaceuticals Incorporated

Microbiotix RNA-dependent RNA polymerase is HCV NS##B, used for the replication of the viral genome.

  • Anti-Infective
  • Genetic Disorder
  • United States
  • Company
  • Product Initiative
  • Target
  • Clinical Trial profile. 525 Trial Title

Part ##.

  • Clinical Trial
  • Cystic Fibrosis
  • Drug Development
  • Genetic Disorder
  • World
  • OCT 04, 2017: TRANSLATE BIO NAMES DANIELLA BECKMAN TO BOARD OF DIRECTORS
  • NOV 06, 2017: TRANSLATE BIO APPOINTS ANN BARBIER, M.D., PH.D., AS CHIEF MEDICAL OFFICER

SR One Ltd.

  • Genetic Disorder
  • Pharmaceutical
  • Therapy
  • United States
  • Company
  • Clinical Trial profile. 49 Trial Title

The prominent features of this report are - ##.

  • Anti-Infective
  • Genetic Disorder
  • Pharmaceutical
  • World
  • Product Initiative
  • OCT 04, 2017: TRANSLATE BIO NAMES DANIELLA BECKMAN TO BOARD OF DIRECTORS
  • NOV 06, 2017: TRANSLATE BIO APPOINTS ANN BARBIER, M.D., PH.D., AS CHIEF MEDICAL OFFICER

SR One Ltd.

  • Genetic Disorder
  • Pharmaceutical
  • Therapy
  • United States
  • Company
  • STEM CELL THERAPY FOR HEMATOLOGICAL DISORDERS AND PRIMARY IMMUNE DEFICIENCY - DRUG PROFILE

The molecules developed by Companies in Pre-Registration, Filing rejected/ Withdrawn, Phase III, Phase II, Preclinical and Discovery stages are ##, ##, ##, ##, ## and ## respectively.

  • Genetic Disorder
  • Pharmaceutical
  • United States
  • Product Initiative
  • ADMA Biologics, Inc.
  • QRX-075 - DRUG PROFILE
  • ARCH BIOPARTNERS INC

It uses technologies including magnetovax and SNIM RNA.

  • Genetic Disorder
  • United States
  • Company
  • Product Initiative
  • Galapagos NV
  • ARCTURUS THERAPEUTICS EXPANDS RESEARCH AGREEMENT WITH CYSTIC FIBROSIS FOUNDATION THERAPEUTICS
  • PROQR THERAPEUTICS ENTERS INTO LICENSING AGREEMENT WITH CYSTIC FIBROSIS FOUNDATION

It uses RNA therapeutics platform to develop all types of RNA medicines including small interfering RNA, messenger RNA, antisense RNA, microRNA, and gene editing therapeutics.

  • Cystic Fibrosis
  • Genetic Disorder
  • Therapy
  • United States
  • Proteostasis Therapeutics, Inc.
  • AUG 29, 2017: PROQR COMPLETES DOSING OF CYSTIC FIBROSIS PATIENTS IN QR-010 PHASE 1B TRIAL
  • JUN 08, 2017: PROQR TO PRESENT QR-010 DATA AT THE EUROPEAN CYSTIC FIBROSIS SOCIETY CONFERENCE AND PROVIDES AN UPDATE ON THE ONGOING PHASE 1B TRIAL

ONE OF THE MOST INTERESTING IS AN RNA PROCESS USED IN NATURE CALLED A TO I EDITING OR ADAR.

  • Genetic Disorder
  • Pharmaceutical
  • Therapy
  • United States
  • ProQR Therapeutics N.V.
  • Target

The prominent features of this report are - ##.

  • Clinical Trial
  • Genetic Disorder
  • World
  • Product Initiative
  • Horizon Pharma plc
  • STEM CELL THERAPY FOR HEMATOLOGICAL DISORDERS AND PRIMARY IMMUNE DEFICIENCY - DRUG PROFILE

No clinically significant food effect was observed (Study-##).

  • Genetic Disorder
  • Infectious Disease
  • Therapy
  • United States
  • Product Initiative

The prominent features of this report are - ##.

  • Clinical Trial
  • Genetic Disorder
  • Therapy
  • World
  • Product Initiative

It uses the enzyme reverse transcriptase to produce DNA from its own RNA.

  • Genetic Disorder
  • Pharmaceutical
  • North America
  • United States
  • Market Size
  • 3. GDCT ID IS THE UNIQUE ID OF GLOBALDATA'S PHARMA INTELLIGENCE CENTER CLINICAL TRIAL DATABASE
  • Official Title

Hoffmann-La Roche Ltd Bayer AG AmpliPhi Biosciences Corp Source: GlobalData' s Pharma Intelligence Center Clinical Trials Database (*)Trials are taken for a time period of 1995 - 2018 2018 trials are taken as of June 2018 ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ##

  • Clinical Trial
  • Genetic Disorder
  • Therapy
  • World
  • Product Initiative
  • Official Title
  • 3. GDCT ID is the unique ID of GlobalData's Pharma Intelligence Center clinical trial database

## ## ## ## ## Insmed Inc PARI Medical Holding GmbH Cystic Fibrosis Foundation Humanigen Inc AmpliPhi Biosciences Corp Allergan Plc ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ## ##

  • Clinical Trial
  • Genetic Disorder
  • Therapy
  • World
  • Product Initiative

Amikacin inhibits protein synthesis by binding to the ##S ribosomal subunit to prevent the formation of an initiation complex with messenger RNA.

  • Anti-Infective
  • Genetic Disorder
  • Respiratory Disease
  • Therapy
  • United States

" If golodirsen and casimersen are approved, nearly a third of the boys and young men living with DMD in the United States could benefit from our RNA therapies.

  • Drug Development
  • Genetic Disorder
  • Pharmaceutical
  • World
  • Product Initiative
  • Prominent Drugs

THE PROMINENT FEATURES OF THIS REPORT ARE - ##.

  • Genetic Disorder
  • Neurological Disorder
  • Therapy
  • World
  • Product Initiative
  • Clinical Trial profile. 4 Trial Title
  • Clinical Trial profile. 535 Trial Title

QR-## is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F##del mutation.

  • Cystic Fibrosis
  • Drug Development
  • Genetic Disorder
  • Pharmaceutical
  • World

Completed Phase IV Interventional doxycycline hyclate University of Nottingham ## Jul 2009 ## Jan 2013 ## ## ## ## ## ##S Ribosomal Subunit (##S RNA) Inhibitor ##S Ribosomal Subunit (##S RNA) Clinical Trial profile. ## Trial Title Official Title Trial Identifier Secondary ID(

  • Genetic Disorder
  • Neurological Disorder
  • World
  • Product Initiative
  • Novartis AG
  • Clinical Trial profile. 13 Trial Title

The prominent features of this report are - ##.

  • Clinical Trial
  • Cystic Fibrosis
  • Genetic Disorder
  • Respiratory Disease
  • Aradigm Corporation