1 But with this shock will come opportunities to continue the progress that has been made, including in the field of neuromuscular diseases, where Califf’s tenure leading the FDA in 2016-2017 ...

While much of the research and discussion around gene therapies for neuromuscular diseases has focused on pediatric patients, this scope is set to widen, said Emma Ciafaloni, MD, FAAN, professor ...

"We remain dedicated to building broader awareness of the devastating impact of rare, progressive neuromuscular disorders including DMD, DM1 and FSHD as well as rare cardiomyopathies and to ...

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin activator, as a promising universal ...

Clinical trials provide an opportunity for patients with rare genetic neuromuscular disorders to access cutting-edge gene modifying treatments before they reach the NHS, as well as give hope to UK ...

Spinal muscular atrophy (SMA) is a group of genetic disorders that causes a loss of motor nerve cells and muscle atrophy. There are five types of SMA relating to age of onset and symptom severity.

We've identified the following companies as similar to LoopUp Group PLC because they operate in a related industry or sector. We also considered size, growth, and various financial metrics to ...

Aside from muscular dystrophy, other examples of rare diseases include hemophilia, an inherited blood disorder that causes excessive bleeding and bruising; phenylketonuria, an inherited disorder ...

The LifeArc and MDUK-funded Translational Rare Disease Centre to Treat Mitochondrial Diseases (LAC-TreatMito-UK) was established in September 2024 to deliver a national platform to translate novel ...

Biplane cineangiocardiography and simultaneous left ventricular pressures were utilized for data acquisition. Normal pressure-volume loops demonstrated only small changes in left ventricular volume ...