We are very excited to announce our conference is going ahead again this year #SaveTheDate, registration opens next week. Stand by to hear our line of of guest speakers!

Well done ⁦Proud Irishman⁩ ⁦Nev Ross⁩ for years of advocacy to ensure that Family carers set to qualify for state pension from 2024 | Business Post businesspost.ie/politics/famil…

Delighted to see my friend Andrew is going to do the Dublin marathon again to help #TreatCureMSD idonate.ie/fundraiser/And… #iDonate_ie If you have something to donate I would be grateful

I am very honoured to be awarded with the WORLDSymposium 2024 Patient Advocate Leader (PAL) Award. More here: tinyurl.com/PAL24award Its the largest Lysosomal conference in the World with approx 3K attending annually. To all that have helped on our journey so far, thank you 😊

Dr. Lars Schlotawa has moved mountains in pursuit of therapies for MSD. We have been blessed in finding such a wonderful doctor and scientist and soon to be Professor. #TreatCureMSD

A brilliant visual abstract of a new publication on biomarkers for Multiple Sulfatase Deficiency. Their reproducibility & our confidence that this informs us on the worst affected biomarkers to look at gives me great confidence that they will help clinical trials #TreatCureMSD

We are very proud to announce that researchers have developed a valuable new induced pluripotent stem cell (iPSC) model of MSD. MSD Action Foundation supported this work with grants to Children's Hospital of Philadelphia. tinyurl.com/MSDiPSC Rebecca Ahrens-Nicklas Vi Pham 1/4

These human cells can be converted into the key cell types that are affected in patients with MSD. For the first time the team identified that iPSC-derived neurons show more hallmarks of MSD, including lysosomal stress, GAG accumulation & reduced enzyme activity, 2/4

compared to undifferentiated cells. This suggests neurons are more sensitive to MSD-induced stress than other cell types. MSD neurons showed defects in early development & differentiation. This provides insights into why patients with MSD have such severe neurologic disease.3/4

A huge thank you to the prinicipal investigators Rebecca Ahrens-Nicklas & Beverly Davidson & all of the authors for your very important work. A special mention to Vi Pham for your hard work on this project & to the director of the pluripotent stem cell core Deborah French at CHOP

Here is a link to the publication which its really important as it can, will & is leading to further MSD research that needs to be done in order to move towards therapies. tinyurl.com/MSDiPSC

Brilliant News Les martin & @DonnellyStephen

Amazing results so far. We were delighted to see this WORLDSymposia in San Diego. Thanks to HealthResearchBoard Health Research Charities Ireland - HRCI for helping to support this superb research project

Great to see results in San Diego from another MSD Action Foundation funded project. If I take off my MSD parent hat & I put on my scientific hat, the future of MSD research is very exciting!

Thank you Xinying Hong from Children's Hospital of Philadelphia Research Institute for presenting your excellent poster at #WORLDSymposium, in San Diego. Your superb biomarker research will make an impact for MSD patients & related MPS conditions #TreatCureMSD

I am very happy to share this video. Thank you to everyone who helped in some way, be it big or small. Much done, more to do. #TreatCureMSD youtu.be/zXMDyJ0bX3c?si…

Can I please ask you to nominate us '20100957- Multiple Sulfatase Deficiency' for £5k in the health section. One nomination per person. Today is the last day, thank you in advance movementforgood.com

Wonderful news 👏

I am so sad to be sharing this news 💔 Our sunshine has passed rip.ie/death-notice/d…

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