16 February 2024
Associate Professor Carlo Rinaldi, a Consultant Neurologist and Clinician Scientist at the Department of Paediatrics, has been awarded the prestigious UKRI MRC Senior Clinical Fellowship for 2024.
31 January 2024
The Department of Paediatrics at the University of Oxford is celebrating the five-year anniversary of a partnership with MDUK. Founded in January 2019, the MDUK Oxford Neuromuscular Centre was set up to drive the development of new therapies and treatments and increase the capacity of clinical trials for muscle wasting and weakening conditions in the UK.
30 August 2023
Scientists at the University of Oxford are running a study at participating hospitals in the Thames Valley and Wessex regions, offering parents the ability to have their newborn baby’s blood tested for Spinal Muscular Atrophy (SMA), in addition to the nine other conditions that are already tested for.
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.
3 December 2021
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.
23 August 2021
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.
9 December 2020
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.
12 August 2020
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.