CureSPG50
@CureSPG50To help children affected by the SPG50 disease
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We want to thank The Hospital for Sick Children for saving my son and truly going above and beyond. We moved mountains together, and hopefully, the treatment we created for Michael will turn out to be a legacy for others with rare diseases, proving that nothing is impossible!
In 2022, 4-year-old Michael received an individual gene therapy at SickKids for #SPG50, an ultra-rare neurodegenerative condition. Today, #SKResearch shows Michael’s progress & the potential impact his clinical trial holds for genetic medicine.🧬 Read ➡️ bit.ly/3La4mBv
Families step up to find gene therapies for diseases too rare for research firms theglobeandmail.com/canada/article…
Blessed to work with this incredibly talented team @BostonChildrens @DEF_Lab_HMS & fantastic families on #raredisease #movementdisorders #HSP. 🙏🏽 @Kasey_47 @CureAP4 for supporting our natural history study (clinicaltrials.gov/study/NCT04712…) & so much more. Teamwork makes the dream work…
Young onset Parkinson's and Charcot Marie Tooth together? Possible? YES. It is called 'FIG 4' and this is the 6th case reported by Silva and colleagues and it was levodopa responsive ~50% improvement in UPDRS. Pallidal hypointensities suggestive of iron deposition. CT scan…
Thank you so very much to @FundaColumbus ⚽️@RealSociedad , @bayer04fussball ⚽️ and all fans that showed up to save children affected by @CureSPG50 like my son. We definitely are not alone!
Some big football/soccer/fútbol/Fussball fans on our #raredisease research team 🇪🇸🇩🇪🇺🇸! Team work makes the dream work! @DariusFakhari @JEAlecu @ScheffoldM @amyyxt @M_Ziegler @cathmjordan @B_Brechmann @h_rosengarten2 @RealSociedad @DFB @Bundesliga_DE @Bundesliga_EN @LaLiga
Thank you for allowing me to tell my story and to thank the hero’s that work at the NIH!
What an incredible story from Terry Pirovolakis, whose son’s journey started @CureSPG50 His tireless efforts to find a cure led them to a number of partners including @NINDSnews and ultimately the targeted gene therapy needed to help save his son. #research #NINDSnonprofitforum
¡𝐘𝐚 𝐚 𝐥𝐚 𝐯𝐞𝐧𝐭𝐚 𝐥𝐚𝐬 𝐞𝐧𝐭𝐫𝐚𝐝𝐚𝐬 𝐩𝐚𝐫𝐚 𝐞𝐥 𝐚𝐦𝐢𝐬𝐭𝐨𝐬𝐨! ⚽️ @RealSociedad - @bayer04_es ⚽️ 🎟️ Entradas a 20€, 10€ para menores de 14 años y gratis para socios: ticketing.realsociedad.eus/#/open/checkou… 🫂 Lucharemos unidos. ¡𝐌𝐞́𝐭𝐞𝐥𝐞 𝐮𝐧 𝐠𝐨𝐥 𝐚𝐥 𝐒𝐏𝐆𝟓𝟎!
An incredible event to save children affected by SPG50! If you can make it, awesome reach out and we will have an amazing time together. If you cannot attend buy a ticket anyways to support this amazing cause!
¡𝐘𝐚 𝐚 𝐥𝐚 𝐯𝐞𝐧𝐭𝐚 𝐥𝐚𝐬 𝐞𝐧𝐭𝐫𝐚𝐝𝐚𝐬 𝐩𝐚𝐫𝐚 𝐞𝐥 𝐚𝐦𝐢𝐬𝐭𝐨𝐬𝐨! ⚽️ @RealSociedad - @bayer04_es ⚽️ 🎟️ Entradas a 20€, 10€ para menores de 14 años y gratis para socios: ticketing.realsociedad.eus/#/open/checkou… 🫂 Lucharemos unidos. ¡𝐌𝐞́𝐭𝐞𝐥𝐞 𝐮𝐧 𝐠𝐨𝐥 𝐚𝐥 𝐒𝐏𝐆𝟓𝟎!
.@FNIH_Org intros new model for #GeneTherapy to get treatments to patients faster. @msstaceyerin blogs about #BGTC bringing hope to people with #RareDiseases @US_FDA @https://www.signalsblog.ca/right-turn-promising-model-for-rare-diseases-in-bespoke-gene-therapy-consortium/
Today on the #SickKidsVS Podcast: 5-year-old Michael is one of less than 100 people in the world with #SPG50, an ultrarare genetic disease. In 2022, a SickKids team dosed Michael with the first-ever #genetherapy in an unprecedented #clinicaltrial. Listen: bit.ly/42z5ghk
Sometimes in life you get to meet true hero’s! I am blessed to have met and know these amazing Drs. and scientists saving children…. Thank you for what you do and for giving families like ours hope!
A research team including #NICHD scientists from @BonifacinoJuan’s lab has developed a potential #GeneTherapy for hereditary spastic paraplegia 50 (SPG50), a rare childhood-onset neurodegenerative disorder. bit.ly/41CRQAS #RareDisease @UTSWBrain @CureSPG50
Thanks to your incredible support, Michael was treated with the Gene Therapy that we were able to create. However, what you may not be aware of is that we used the funds raised to create two additional treatments for SPG50. Over the past 12 months, we ha… instagr.am/reel/CqRWmSXON…
Out now: Our paper describing the preclinical development of AAV9/AP4M1 - the first #genetherapy for #hereditaryspasticparaplegia.👇🏽was led by Xin Chen & Steven Gray @UTSWBrain Thankful for this collaboration @UTSWNews @BostonChildrens @NIH @CureSPG50 jci.org/articles/view/…
--Stop, Read & Share Please!-- If you're an out-of-the-box thinker with experience in managing pre-clinical and clinical gene therapy projects and you're living in California, we want you on our team! linkedin.com/jobs/view/3524…
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CSNK2A1 Foundation
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