For people with DM1, this leads to symptoms like progressive muscle weakness ... A variation really stood out to me that we see in rare neuromuscular disease. 'This is how we started connecting ...

The researchers believe this class of drugs, widely used to treat a number of cardiovascular diseases ... 1 (DM1) and further shows that calcium channel blockers can reverse these symptoms ...

Entrada has also applied its cyclic cell-penetrating peptides to the treatment of myotonic dystrophy type 1 (DM1), a rare disease that affects about 40,000 people in the U.S. and causes symptoms ...

DM1 patients can suffer from various manifestations of disease including myotonia, or a temporary rigidity due to the inability to relax muscles, muscle weakness, cardiac abnormalities ...

Similarly, people with DM1 have a TRE in the DMPK gene. "A variation really stood out to me that we see in rare neuromuscular diseases," says ... of causing autistic symptoms in children with ...

The lead indication is Myotonic Dystrophy Type 1 (DM1), a rare ... and these carriers will have symptoms of the disease, unlike autosomal recessive disorders, where two copies of the mutated ...

Download a PDF of the Plain Language Summary. With a median follow-up of 8.4 years, T-DM1 sustained the improvement in invasive disease–free survival over trastuzumab (unstratified hazard ratio ...

The Muscular Dystrophy Association highlights two types of Myotonic MD: DM1 ... disease and help prevent complications. People should see a doctor if they or their child develop signs or symptoms ...

Consistent with earlier reports from the study, the 7-year invasive disease-free survival (iDFS) improved from 67.1% with trastuzumab to 80.8% with T-DM1. Grade ≥3 adverse events occurred more ...

Trastuzumab emtansine (T-DM1) demonstrates sustained improvement in invasive disease-free survival and reduced the risk for death in human epidermal growth factor receptor 2 (HER2)-positive breast ...

Mis-splicing is the underlying cause of DM1 pathology, and we believe the mean ... s potential to address the underlying cause of disease," said James McArthur, PhD, President and CEO of PepGen.