Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

Diagnosed with Duchenne muscular dystrophy (DMD) at three years old, Dr Jon Hastie grew up never thinking about the future – until one day he discovered he actually had one. Now in his 30s ...

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD), becoming the first non-steroidal option for patients with all ...

Phil Lambert, Ph.D., chief scientific officer of Satellos Bioscience Inc., explains the mechanism of action of the company's experimental Duchenne muscular dystrophy drug, SAT-3247, and the thinking ...

Dr Milad Shadrooh AKA The Singing Dentist. When to start brushing, how to cope with resistance, fears of the dentist… Parents often have lots of burning questions about how to look after ...

Diabetes may play a role in your weight, including weight loss, when you have type 1 or type 2 diabetes. This can be a symptom of unmanaged or undiagnosed diabetes, but it can also be due to other ...

The dentist needed to redo the filling due to pain and it was swollen, etc. This was also not covered and I have to pay as you can only get a filling redone after two years. Lastly, a crown was ...

Avoid the Rancho office at all costs. This location is a masterclass in mismanagement, unprofessionalism, and complete disregard for patient care. I began an implant procedure in October 2024.

For our high-throughput screening campaign, we utilized an NSC34 motor neuron cell line expressing an SMN2 minigene reporter. A pair of SMN2 matched reporter constructs were designed such that one ...