The Limb-Girdle Muscular Dystrophy drugs in development market research report provides comprehensive information on the therapeutics under development for Limb-Girdle Muscular Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Limb-Girdle Muscular Dystrophy. Buy the report here.

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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Limb-Girdle Muscular Dystrophy and features dormant and discontinued products.

GlobalData tracks 22 drugs in development for Limb-Girdle Muscular Dystrophy by 15 companies/universities/institutes. The top development phase for Limb-Girdle Muscular Dystrophy is preclinical with 13 drugs in that stage. The Limb-Girdle Muscular Dystrophy pipeline has 21 drugs in development by companies and one by universities/ institutes. Some of the companies in the Limb-Girdle Muscular Dystrophy pipeline products market are: Sarepta Therapeutics, Atamyo Therapeutics and MyoPax.

The key targets in the Limb-Girdle Muscular Dystrophy pipeline products market include Gamma Sarcoglycan (35 kDa Dystrophin Associated Glycoprotein or SGCG), Fukutin Related Protein (Ribitol 5 Phosphate Transferase or FKRP or EC 2.4.2.), and Calpain 3 (Calcium Activated Neutral Proteinase 3 or Calpain L3 or Calpain p94 or Muscle Specific Calcium Activated Neutral Protease 3 or New Calpain 1 or CANP3 or CAPN3 or EC 3.4.22.54).

The key mechanisms of action in the Limb-Girdle Muscular Dystrophy pipeline product include Gamma Sarcoglycan (35 kDa Dystrophin Associated Glycoprotein or SGCG) Activator with two drugs in Phase I. The Limb-Girdle Muscular Dystrophy pipeline products include six routes of administration with the top ROA being Intravenous and seven key molecule types in the Limb-Girdle Muscular Dystrophy pipeline products market including Gene Therapy, and Small Molecule.

Limb-Girdle Muscular Dystrophy overview

Limb-girdle muscular dystrophy encompasses a spectrum of disorders impacting the voluntary muscles encircling the hips and shoulders, exhibiting equal prevalence in males and females. The majority of these dystrophies follow an autosomal recessive inheritance pattern, although some rarer forms adhere to an autosomal dominant model. With over 20 recognized types, this condition arises from genetic material (DNA) alterations and can manifest at any point in life. Its characteristic feature is progressive muscle weakness, affecting daily activities. Diagnosis involves genetic testing, clinical evaluation, and may require interventions to manage symptoms and enhance quality of life, given the absence of a cure.

For a complete picture of Limb-Girdle Muscular Dystrophy’s pipeline drug market, buy the report here.

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GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.