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Dr Matthew Snape leads a study investigating if giving small amounts of oral insulin to babies can prevent type 1 diabetes, thus allowing mothers to protect their children from injecting insulin.
Novartis Global Scholar Program award for Neuromuscular team
Awards & Appointments Department Neuromuscular Diseases Research
6 March 2024
Many congratulations to Associate Professor Carlo Rinaldi and Senior Postdoctoral Scientist Antonio Garcia-Guerra, who have been awarded the Novartis Global Scholar Program (NGSP) for 2023.
Associate Professor Carlo Rinaldi awarded prestigious Clinical Fellowship
Awards & Appointments Department Neuromuscular Diseases Research
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.
MDUK Oxford Neuromuscular Centre celebrates five year anniversary
Clinical Trials Department Neuromuscular Diseases Research
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.
New study to test babies for Spinal Muscular Atrophy
Children's Health Clinical Trials Neuromuscular Diseases Research
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.
AR cooperates with SMAD4 to maintain skeletal muscle homeostasis
Neuromuscular Diseases
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
Neuromuscular Diseases Research
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.
Promising discovery for treatment of neuromuscular diseases
Neuromuscular Diseases Publication Research
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.
Why we must expand newborn screening
Neuromuscular Diseases
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
Awards & Appointments Neuromuscular 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.
Enriching Engagement (Round 2) awardees announced
Awards & Appointments Neuromuscular Diseases Public Engagement
25 August 2020
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
Neuromuscular Diseases Publication
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.