This year, the 2022 MDA Clinical & Scientific Conference began March 13, 2022 and is lasting through March 16, 2022. During the conference, stakeholders will discuss a variety of clinical achievements and research advancements associated with NMD. Biotechnology company NeuBase Therapeutics, Inc. (“NeuBase”) will be presenting twice during MDA 2022. According to Yahoo! Finance, NeuBase’s presentations will center around precision medicines for myotonic dystrophy type 1 (DM1) created using its proprietary PATrOL™ platform.
Within the Company’s poster presentation, NeuBase discusses the pharmacology, tolerability, and biodisributive properties of an investigational genetic treatment for those with DM1. Their next presentation, delivered orally on the morning on March 16th, will explore preclinical findings from mice models of myotonic dystrophy type 1 treated with therapeutics developed on the proprietary platform. Currently, the company is developing NT-0200 as a potential therapeutic candidate for DM1. As NeuBase explains on its website:
Preclinical data demonstrates NeuBase’s drug candidate maintains similar DMPK protein levels to untreated patient-derived cell lines and rescues splicing across significantly mis-spliced muscle mRNA transcripts in the HSALR mouse model.
These findings will be expanded upon during the MDA 2022 presentations. To learn more about MDA 2022 and the presentations, head here.
About Myotonic Dystrophy
Myotonic dystrophy is a rare genetic disorder characterized by progressive muscle wasting and weakness. It is considered the most common form of adult-onset muscular dystrophy. There are two subtypes of myotonic dystrophy: type 1 (DM1) and type 2 (DM2). An abnormally expanded section on DMPK causes DM1, while an abnormally expanded section on ZNF9 causes DM2. Typically, the characteristics and symptoms of both forms overlap. However, DM2 is considered milder than myotonic dystrophy type 1. Additionally, DM1 more often affects the lower legs, hands, face, and neck; DM2 typically affects the hips, elbows, shoulders, and neck. Other symptoms include:
- Myotonia (inability to relax muscles at will)
- Difficulty swallowing
- Gallstones
- Muscle pain
- Cataracts
- Pregnancy and labor complications
- Type 2 diabetes
- Difficulties with speech and hearing
- Vision fatigue
- Learning disabilities and/or cognitive impairment
- Difficulty breathing
- Constipation
- Abnormal heart rate
- Weakness of the gastrointestinal tract muscles, heart muscle, breathing muscles, and voluntary muscles
Learn more about myotonic dystrophy.
