Lafora Disease (Unverricht Disease) drugs in development by stages, target, MoA, RoA, molecule type and key players, 2023

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 Lafora Disease (Unverricht Disease) and features dormant and discontinued products.

GlobalData tracks seven drugs in development for Lafora Disease (Unverricht Disease) by five companies/universities/institutes. The top development phase for Lafora Disease (Unverricht Disease) is preclinical with five drugs in that stage. The Lafora Disease (Unverricht Disease) pipeline has six drugs in development by companies and one by universities/ institutes. Some of the companies in the Lafora Disease (Unverricht Disease) pipeline products market are: Ionis Pharmaceuticals, Taysha Gene Therapies and ANLBio.

The key targets in the Lafora Disease (Unverricht Disease) pipeline products market include Adenosine Monophosphate Activated Protein Kinase ([Hydroxymethylglutaryl CoA Reductase (NADPH)] Kinase or AMPK or EC 2.7.11.31), Glycogen Starch Synthase (GYS1 or EC 2.4.1.11), and Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20).

The key mechanisms of action in the Lafora Disease (Unverricht Disease) pipeline product include Adenosine Monophosphate Activated Protein Kinase ([Hydroxymethylglutaryl CoA Reductase (NADPH)] Kinase or AMPK or EC 2.7.11.31) Activator with one drug in Preclinical. The Lafora Disease (Unverricht Disease) pipeline products include two routes of administration with the top ROA being Intrathecal and four key molecule types in the Lafora Disease (Unverricht Disease) pipeline products market including Gene Therapy, and Antisense Oligonucleotide.

Lafora Disease (Unverricht Disease) overview

Unverricht-Lundborg disease (EPM1) a rare and fatal genetic neurodegenerative disorder that causes progressive myoclonic epilepsy. It is caused by mutations of the cystatin B gene. It typically presents in adolescents with new onset seizures along with symptoms such as lack of motor coordination, impaired mental function, and depression. It is diagnosed with a skin biopsy that shows Lafora bodies in the cells of a patient’s sweat ducts. Anti-seizure medications (ASMs) are used to treat seizures and myoclonus.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

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.