In recent years, a growing understanding of myotonic dystrophy type 1 (DM1)—an often fatal ... the cause of DM1 is genetic. An alteration in the causative gene, dystrophia myotonica protein ...

People with myotonic dystrophy type 1 (DM1), the most common adult-onset ... In the current study published in Human ...

EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Medically reviewed by Jane Kim, MD Myotonic muscular dystrophy, which is sometimes called myotonic dystrophy, is a type of ...

San Diego-based Kate is focused on preclinical programs aimed at Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD) and myotonic dystrophy type 1 (DM1). These programs have ...

Rare genetic disorders affect approximately 1 in 10 people worldwide, meaning over 350 million individuals are living with these conditions. Despite their rarity, there are over 7,000 identified rare ...

Avidity Biosciences' AOC platform shows promising results for genetic diseases, making it a strong buy with potential upside ...

Novartis has reached an agreement to take control of Kate Therapeutics, a developer of gene therapies ... facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1), as well as ...

Arrakis' oral therapies targeting disease-driving RNA in myotonic dystrophy have shown promise in preclinical studies.

Myotonic Dystrophy Pipeline Report Introduction 2. Myotonic Dystrophy Pipeline Report Executive Summary 3. Myotonic Dystrophy ...

Kate Therapeutics is a preclinical-stage biotechnology company that focuses on adeno-associated virus (AAV)-based gene ... dystrophy, facioscapulohumeral dystrophy and myotonic dystrophy type 1 ...