Search results (7)
« Back to NewsAR cooperates with SMAD4 to maintain skeletal muscle homeostasis
18 May 2022
Skeletal muscle, which accounts for over 40% of the total mass in healthy individuals, plays a central role in maintenance of organismal homeostasis. Conversely, muscle atrophy upon acute and chronic conditions, ranging from genetic muscular dystrophy to critical illnesses, cachexia and sarcopenia, significantly correlates with levels of disability and is an important predictor of mortality. Despite the urgent medical need, treatments able to efficiently counteract muscle loss are lacking due to an incomplete understanding of the underlying intricate molecular mechanisms of regulation.
Angelman syndrome: first patient to receive potential therapy in Oxford
3 December 2021
Neuromuscular Diseases Research
Things that seemed impossible, only a few years ago, are happening today. The first patient in Europe and one of the first in the world was injected with a potential treatment, GTX-102, in a phase I/II clinical trial in Oxford.
Promising discovery for treatment of neuromuscular diseases
23 August 2021
Neuromuscular Diseases Publication Research
Research led by Carlo Rinaldi and Catheryn Lim discovered that a naturally-occurring isoform of an androgen receptor can be used in therapy for spinal and bulbar muscular atrophy, and shows potential for other diseases.
Why we must expand newborn screening
6 May 2021
Early diagnosis is of primary importance both to obtain the best effect of innovative medications and to accelerate their development, writes Professor Laurent Servais.
University spinout PepGen awarded major financing to target Duchenne muscular dystrophy
9 December 2020
Awards & Appointments Neuromuscular Diseases
PepGen, a therapeutics company targeting severe neuromuscular diseases, including Duchenne muscular dystrophy (DMD), has closed a $45 million Series A funding round led by RA Capital Management with participation from Oxford Sciences Innovation (OSI), the company’s original seed investor.
Enriching Engagement (Round 2) awardees announced
25 August 2020
Awards & Appointments Neuromuscular Diseases Public Engagement
The awardees for the second round of the University of Oxford’s Enriching Engagement funding scheme have now been announced, including a project from Paediatrics.
Featured publication: Advances in oligonucleotide drug delivery
12 August 2020
Neuromuscular Diseases Publication
Oligonucleotides - short DNA or RNA molecules - have great therapeutic application for a range of diseases. Yet, their potential has not been fully unleashed because of challenges linked to efficient delivery. Read more to find out about recent developments in oligonucleotide modifications, and the platforms that may be used to deliver them to target sites.