Facioscapulohumeral Muscular Dystrophy (FSHD) – Drugs In Development, 2024

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Facioscapulohumeral muscular dystrophy (FSHD) typically manifests with varying degrees of facial, scapular stabilizer, and dorsiflexor muscle weakness. Progression is slow, with about 20% of individuals eventually requiring a wheelchair, though life expectancy remains unaffected. Diagnosis of FSHD1 involves identifying a heterozygous pathogenic contraction of the D4Z4 repeat array on chromosome 4q35 with a chromosome 4 permissive haplotype. FSHD2 diagnosis is established by detecting D4Z4 repeat array hypomethylation on chromosome 4q35 with the same haplotype, linked to heterozygous pathogenic variants in SMCHD1 or DNMT3B. Treatment addresses symptoms, involving physical therapy, orthoses, surgery for scapular fixation, pain management, respiratory function monitoring, and specific interventions for ocular, auditory, and other associated issues.

The Facioscapulohumeral Muscular Dystrophy (FSHD) drugs in development market research report provide comprehensive information on the therapeutics under development for Facioscapulohumeral Muscular Dystrophy (FSHD), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Facioscapulohumeral Muscular Dystrophy (FSHD) and features dormant and discontinued products.

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* Certain sections in the report may be removed or altered based on the availability and relevance of data.

Quick View – Facioscapulohumeral Muscular Dystrophy (FSHD)
Key Targets
· Double Homeobox Protein 4; AP2 Associated Protein Kinase 1; Androgen Receptor; Mitogen Activated Protein Kinase 11; Mitogen Activated Protein Kinase 14; Structural Maintenance Of Chromosomes Flexible Hinge Domain Containing Protein 1; Growth/Differentiation Factor 8; and Transferrin Receptor Protein 1
Key Mechanisms of Action
· Double Homeobox Protein 4 Inhibitor; Androgen Receptor Agonist; AP2 Associated Protein Kinase 1 Inhibitor; Growth/Differentiation Factor 8 Inhibitor; Mitogen Activated Protein Kinase 14 Inhibitor; Mitogen Activated Protein Kinase 11 Inhibitor; and Structural Maintenance Of Chromosomes Flexible Hinge Domain Containing Protein 1 Activator
Key Routes of Administration
· Oral; Parenteral; Intravenous; Intramuscular; and Subcutaneous
Key Molecule Types
· Small Molecule; Gene Therapy; Antisense Oligonucleotide; Antisense RNAi Oligonucleotide; Biologic; Cell Therapy; Monoclonal Antibody; Oligonucleotide; Monoclonal Antibody Conjugated; and Gene-Modified Cell Therapy
Major Companies
· Sanofi; F. Hoffmann-La Roche Ltd; Facio Therapies BV; Fred Hutchinson Cancer Research Center; Fulcrum Therapeutics Inc; Healx Ltd; Kate Therapeutics Inc; Modalis Therapeutics Corp; Myocea Inc; and Nationwide Children's Hospital

Scope

  • Therapeutics in Development: Covering 24 molecules, with 19 developed by companies and the rest by universities/institutes.
  • Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
  • Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
  • Development History: Access the complete research and development history.
  • Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.

Reasons to Buy

  • Holistic insights: Understand the broad spectrum of Facioscapulohumeral Muscular Dystrophy (FSHD) therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
  • Pipeline Exploration: Explore the detailed Facioscapulohumeral Muscular Dystrophy (FSHD) pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
  • Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Facioscapulohumeral Muscular Dystrophy (FSHD) treatments for adaptable decision-making.
  • Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.

Key Players

Altay Therapeutics Inc
Armatus Bio LLC
Arrowhead Pharmaceuticals Inc
Avidity Biosciences Inc
Celularity Inc
Children's National Hospital
Dyne Therapeutics Inc
ECM Therapeutics Inc
Epicrispr Biotechnologies Inc
F. Hoffmann-La Roche Ltd
Facio Therapies BV
Fred Hutchinson Cancer Research Center
Fulcrum Therapeutics Inc
Healx Ltd
Kate Therapeutics Inc
Myocea Inc
Nationwide Children's Hospital
Renogenyx Inc
Sanofi
Sarcomed AB
University of Minnesota
University of Montpellier
Vita Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Overview

Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Drugs Under Development by Companies

Drugs under Development by Universities/Institutes

Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Companies Involved in Therapeutic Development

Drug Profiles

Dormant Projects

Discontinued Drugs

Product Development Milestones

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Drugs under Development by Companies, 2024

Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Stage and Target, 2024

Number of Drugs by Stage and Mechanism of Action, 2024

Number of Drugs by Stage and Route of Administration, 2024

Number of Drugs by Stage and Molecule Type, 2024

Dormant Projects, 2024

Discontinued Drugs, 2024

Figures

List of Figures

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Top 10 Targets, 2024

Number of Drugs by Stage and Top 10 Targets, 2024

Number of Drugs by Top 10 Mechanism of Actions, 2024

Number of Drugs by Stage and Top 10 Mechanism of Actions, 2024

Number of Drugs by Top 10 Routes of Administration, 2024

Number of Drugs by Stage and Top 10 Routes of Administration, 2024

Number of Drugs by Top 10 Molecule Types, 2024

Number of Drugs by Stage and Top 10 Molecule Types, 2024

Frequently asked questions

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