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Hot off the press...critical toxicology work for our U.S. FDA IND application toward a clinical trial testing ASA for #RareDisease #myotwitter Adenylosuccinic Acid Is a Non-Toxic Small Molecule In Vitro and In Vivo mdpi.com/2518800 #mdpipharmaceuticals via MDPI

Excited to see our work featured in Rare Disease Advisor. Congrats Stephanie Kourakis Cara Timpani Dean Campelj PhD & collabs on an insightful paper with huge reach! Research Explores Duchenne Muscular Dystrophy Care of the Past and Present rarediseaseadvisor.com/insights/dmd-i…

Congrats Stephanie Kourakis Cara Timpani Dean Campelj PhD and team...another feature in a recent Expert Point of View by Prof Laurent Servais from Department of Paediatrics on neurodiem bit.ly/_Kourakis-et-a…

Full text here: rdcu.be/dpzHd Institute for Health and Sport, VU Victoria University AIMSS AIMSS Inherited & Acquired Myopathy Program

Very excited for our latest research to be finally out in the world! Loss of endogenous estrogen alters mitochondrial metabolism and muscle clock-related protein Rbm20 in female mdx mice biorxiv.org/content/10.110…

Today on Rare Disease Day, we will host @cureadssl1 in a virtual luncheon to learn about the patient experience and the impact of our research and engagement activities. Please join us @ 2.30pm AEST showing your stripes to learn more. Details below. Victoria University #RareDisease

We're excited to announce the second keynote speaker for the 7th VictorianMuscleNetwork Symposium! Join us on Fri, Nov 8th, to hear from Dr. Eppie Yiu from Murdoch Children's Research Institute (MCRI) , alongside Prof. Jeffrey Chamberlain from University of Washington

Registrations and abstract submissions for the 2024 AussieMit conference are open! Join us: 📅 26 – 29 November 📍 Melbourne Museum (Melbourne Museum), Melbourne, VIC Learn more: aussiemit.com.au/register/ #MitochondrialDisease #Mitochondria #Mito

Really excited that our work assessing the therapeutic effect of moderate-term dimethyl fumarate on dystrophic (mdx) muscle is out in the world! Thanks to funding from Muscular Dystrophy Association for making this study possible! #Duchenne Victoria University AIMSS Inherited & Acquired Myopathy Program biorxiv.org/content/10.110…

Highlighting our 2nd pub of the week to be out in the world! Got to probe a new proteomics dataset & we identified 2 potential cachexia biomarkers in an anti-leukemia mouse model. Nice work led by Emma Rybalka & Dean Campelj PhD. #myotwitter AIMSS Inherited & Acquired Myopathy Program biorxiv.org/content/10.110…

Come visit the Cure Adssl1 stand at #WMSPrague2024. We are looking for patients to join our disease registry and collaborators to join our research consortium and mission to find a treatment for ADSS1 myopathy. ⁦World Muscle Society⁩ #cureadssl1 linkedin.com/posts/emma-ryb…

🎤 Exciting update! The VictorianMuscleNetwork will feature a panel discussion with experts in the field: Dr. William Roman William Roman Dr. Avnika Ruparelia Dr Avnika Ruparelia A/Prof. Ben Parker Benjamin Parker Prof. Jeffrey Chamberlain Dr. Eppie Yiu Join us for an insightful conversation on Nov 8th

So very thrilled to be part of this NHMRC Ideas Grant! Cannot wait to work with this talented team to develop the first treatment for ultra-rare ADSS1 myopathy!

Over 300 million people worldwide are living with a #raredisease. On #RareDiseaseDay2025, I thought I would share our paper on ultra-rare disease, adenylosuccinate synthetase 1 myopathy pubmed.ncbi.nlm.nih.gov/39593137/.

Proud as punch to be part of Stephanie Kourakis's first author #RedoxBiology paper - she's kicking goals in her PhD! Repurposed Nrf2 activator dimethyl fumarate rescues muscle inflammatio... sciencedirect.com/science/articl… Emma Rybalka Victoria University #Duchenne

Happy to be part of Dean Campelj PhD's paper which has identified two potential biomarkers of cachexia induced by anti-AML chemotherapy. Emma Rybalka Victoria University onlinelibrary.wiley.com/doi/10.1002/jc…