Myotonic dystrophy type 1 (DM1), a genetic disorder, shares overlapping features with chronic kidney disease, including progressive muscle wasting, whole-body insulin resistance and impaired energy ...

Cellular models used to search for new therapies for myotonic dystrophy type 1 do not usually take into account the diversity of subtypes presented by patients. A study led by researchers from the ...

Using brain organoids, researchers discover mutational commonalities between muscular dystrophy type 1 and Rett syndrome, suggesting the potential of a similar treatment for both. Myotonic dystrophy ...

AOC 1001 consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 conjugated with a small interfering RNA. The Food and Drug Administration (FDA) has granted Fast Track ...

Dyne expects a potential U.S. launch of z-basivarsen in H1 2028, assuming FDA grants Priority Review and approval is received on the anticipated timeline. Dyne also continues to pursue approval ...

September 6, 2007 — A new study using a mouse model of myotonic dystrophy type 1 (DM1) has revealed an early step in the process by which a mutant DM gene leads to cardiac defects. Found in more than ...

Spain’s ARTHEx Biotech has developed a dual-mechanism approach to treating the most common adult-onset muscular dystrophy, myotonic dystrophy type 1, setting it apart in a burgeoning blockbuster ...

Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic complications ...

Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function. Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular ...

Dyne Therapeutics announces FDA Fast Track designation for DYNE-101, targeting myotonic dystrophy type 1 with potential accelerated approval in 2026. Dyne Therapeutics has announced that its ...

Scientists from The Hospital for Sick Children (SickKids) and University of Las Vegas Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic condition ...

Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...