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

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 ...

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 ...

They claim to cover 80% of "preventative care" services for out-of-network dentists. This appears on term sheets they advertise to the public, on their "member benefits document" available in ...

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 ...