Dystrophin-associated protein complex

First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy Presented at International Myology 2024 Congress

Retrieved on: 
Tuesday, April 23, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Safety, Myology, Dystrophin-associated protein complex, CPK, NSAA, Immunohistochemistry, Duchenne, Duchenne muscular dystrophy, Disease, Patient, Ageing, Pharmaceutical industry

Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.

Key Points: 
  • Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.
  • The trial combines phases I/II/III with a dose escalation phase, followed by a pivotal phase at the selected dose.
  • The trial has been approved by French and UK health authorities and includes ambulant boys aged 6 to 10 suffering from Duchenne Muscular Dystrophy.
  • Note that the dose chosen is lower than those used in other gene therapy trials for Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy 2022: Epidemiology, Etiology, Pathophysiology, Symptoms, Diagnosis and Treatment Guidelines - ResearchAndMarkets.com

Retrieved on: 
Friday, February 18, 2022
Biotechnology, Pharmaceutical, Health, DMD, Muscle, Relaxation, Patient, Muscle contraction, Epidemiology, Mortality, Cardiomyopathy, Compound annual growth rate, HIV disease progression rates, Phenotype, Review, Female, Therapy, Dystrophinopathy, X-linked recessive inheritance, Duchenne muscular dystrophy, Protein, Genetic disorder, Survival, Dystrophin-associated protein complex, Steroid, D, Disease, DPC, Industry, Weakness, Diagnosis, Dystrophin, Exercise-associated muscle cramps, Life, CAGR, Dentistry, Pharmaceutical industry

The "Duchenne Muscular Dystrophy - Opportunity Assessment and Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Duchenne Muscular Dystrophy - Opportunity Assessment and Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.
  • Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin.
  • Greatest unmet needs in the DMD space pertain to disease progression rate, access to treatment for specific patient segments, and cost of therapies.
  • Overview of DMD including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.