Report Type : Therapy Reports
Report Category : Genetic Disease
Report Subcategory : Duchenne Muscular Dystrophy
Price : 2999.00
Date : February 2023
Duchenne Muscular Dystrophy : Global Market Landscape till 2030 - Epidemiology, Drugs, Trials, Technology, Companies, News, Deals, Patents, Drug Sales & Forecast, Future Events, DMF, and Manufacturing
Duchenne Muscular Dystrophy:
Duchenne Muscular Dystrophy, often abbreviated as DMD, is a progressive and severe muscle-wasting condition. Individuals diagnosed with DMD, a form of muscular dystrophy, face significant challenges. By the age of 12, many are reliant on wheelchairs due to muscle degeneration and also encounter social isolation. The condition complicates even the simplest daily activities and, as it progresses, critically affects the heart and respiratory muscles. Duchenne is classified under muscular dystrophies, genetic diseases marked by muscle weakness, with Duchenne being notably the most severe due to the absence of dystrophin, a protein essential for muscle integrity.
Prevalence:
Duchenne impacts around 15,000 individuals in the United States, predominantly males, and over 300,000 globally. It is a condition that transcends cultural, economic, and social lines, affecting families worldwide.
Cardiac Dysfunction
Adolescence is typically when signs of cardiac dysfunction, a serious disease complication, first become apparent. Nonetheless, maintaining physical activity levels, even among those who are wheelchair-bound, can improve the management of cardiac symptoms.
Advancements in Therapies:
Encouragingly, recent years have seen breakthroughs in gene-based therapies targeting DMD. These advancements include gene replacement techniques, RNA-based therapies, and pharmacological interventions, with antisense-mediated exon skipping emerging as a particularly promising approach for addressing the muscular dystrophy caused by Duchenne.
Top 50 Key Players:
F. Hoffmann-La Roche Ltd
Pfizer Inc
Takeda Pharmaceutical Co Ltd
Asahi Kasei Corp
Biogen Inc
Otsuka Holdings Co Ltd
Astellas Pharma Inc
Mylan NV
Teijin Ltd
Les Laboratoires Servier SAS
Vertex Pharmaceuticals Inc
Sarepta Therapeutics Inc
Lexicon Pharmaceuticals Inc
PTC Therapeutics Inc
FibroGen Inc
Retrophin Inc
Bioleaders Corp
Santhera Pharmaceuticals Holding AG
Acceleron Pharma Inc
Fate Therapeutics Inc
NicOx SA
Capricor Therapeutics Inc
Pluristem Therapeutics Inc
University of Wisconsin Madison
CRISPR Therapeutics
Nationwide Children's Hospital
Genethon SA
Tolerion Inc
University of California Los Angeles
The Hospital for Sick Children
Stem Cells of Arabia
Dystrogen Therapeutics SA
Stanford University
Spinalcyte Llc
MyoGene Bio LLC
InnoBioscience LLC
Axolo Pharma Inc
ImmunoForge Co Ltd
Prothelia Inc
Solid Biosciences Inc
Penn Medicine
University California
Ace Technologies Corp
Biophytis SA
Epirium Bio Inc
Eloxx Pharmaceuticals Inc
Italfarmaco Holding SpA
EryDel SPA
Catabasis Pharmaceuticals Inc
University Children's Hospital Basel
