According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating the company’s experimental treatment AMO-02 as a therapy for congenital myotonic dystrophy. AMO is focused on developing promising treatments for debilitating and serious diseases with urgent unmet medical need and few to no treatment options.
About Myotonic Dystrophy
Myotonic dystrophy is a form of muscular dystrophy. Its onset is later than many other forms, often beginning in the 20s and 30s (though it can begin at any time). In this long-term disorder, the muscle systems contract and are unable to relax. The disease comes in two main forms: type 1, which is usually more severe and linked to mutations affecting the DMPK gene, and type 2, which is caused by mutations of the CNBP gene. When the type 1 form begins at birth, it is often referred to as congenital myotonic dystrophy. Symptoms include progressive muscle weakness, muscle atrophy, heart problems, intellectual disability, cataracts, sleep apnea, and insulin resistance. Men may experience infertility and early balding. Muscle problems often begin in the feet, hands, neck, or face. There are no disease altering therapies for the disorder, and treatment focuses on managing symptoms and complications. Common measures include pacemakers, non-invasive ventilation, and mobility aids such as braces or a wheelchair. The disease is estimated to affect around 30,000 people in the US. To learn more about myotonic dystrophy, click here.
The clinical trial will include both child and adolescent patients with living with the disease.
About AMO-02
AMO-02, also called tideglusib, is an investigational therapy candidate in development as a treatment for congenital myotonic dystrophy, a form that begins in infancy. This drug may also have potential for use in the more common adult-onset variant as well. AMO-02’s mechanism of action is designed to interrupt the disease-causing RNA repeat found in the disease and also prevent kinase GSK3β levels from getting too high.
Hopefully, the results of this trial will be positive and support the approval of this therapy for congenital myotonic dystrophy.
