.Foundation for the National Institutes of Health intros new model for #GeneTherapy to get treatments to patients faster. Stacey Johnson blogs about #BGTC bringing hope to people with #RareDiseases U.S. FDA @https://www.signalsblog.ca/right-turn-promising-model-for-rare-diseases-in-bespoke-gene-therapy-consortium/

¡𝐘𝐚 𝐚 𝐥𝐚 𝐯𝐞𝐧𝐭𝐚 𝐥𝐚𝐬 𝐞𝐧𝐭𝐫𝐚𝐝𝐚𝐬 𝐩𝐚𝐫𝐚 𝐞𝐥 𝐚𝐦𝐢𝐬𝐭𝐨𝐬𝐨! ⚽️ Real Sociedad Fútbol - Bayer 04 Leverkusen ⚽️ 🎟️ 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!

Fundación Columbus Bayer 04 Leverkusen Xabi Alonso Real Sociedad Fútbol Contra el Cáncer en Gipuzkoa Boston Children's Bayer 04 Leverkusen Bayer 04 Leverkusen CureSPG50 Some big football/soccer/fútbol/Fussball fans on our #raredisease research team 🇪🇸🇩🇪🇺🇸! Team work makes the dream work! Darius Ebrahimi-Fakhari @JEAlecu Marlene Scheffold Amy Tam @M_Ziegler Catherine Jordan Barbara Brechmann helena Real Sociedad Fútbol DFB (Verband) BUNDESLIGA Bundesliga English LALIGA

Thank you for allowing me to tell my story and to thank the hero’s that work at the NIH!

Thank you so very much to Fundación Columbus ⚽️Real Sociedad Fútbol , Bayer 04 Leverkusen ⚽️ and all fans that showed up to save children affected by CureSPG50 like my son. We definitely are not alone!

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

Blessed to work with this incredibly talented team Boston Children's Ebrahimi-Fakhari_Lab & fantastic families on #raredisease #movementdisorders #HSP. 🙏🏽 Kasey Edwards CureAP-4 for supporting our natural history study (clinicaltrials.gov/study/NCT04712…) & so much more. Teamwork makes the dream work…

Families step up to find gene therapies for diseases too rare for research firms theglobeandmail.com/canada/article…

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!

Thank you, STAT and Jason Mast, for sharing our story and raising awareness about our family’s journey and Elpida Therapeutics Therapeutics. The road has been long and difficult, but our children are far too important to ever give up on. I hope this story brings hope to families in

Last Rare Disease Day, I found myself urgently trying to reach Senator Sanders and his team, who posed the biggest threat to preserving the Priority Review Voucher (PRV) program. The devastation I felt when Senator Paul, stating that his senatorial obligations had run their

I am deeply honored and grateful to have been selected to speak at the Cell & Gene Therapy Forum today at The White House The event was truly inspiring, bringing together leaders, advocates, mentors, and so many individuals who have supported and continue to support our journey.

Rare Disease In Crisis, Our Leaders Have Failed Us! On April 2nd, 2019, a piece of my soul was taken from me. That was the day my youngest son, Michael, was diagnosed with a rare disease called SPG50. In my darkest moments, friends, family, and strangers from around the world

Thank you all for your messages and the incredible wave of support. The past three months have been the hardest of my life. We’ve lost all avenues of funding, and years of hard work preparing for our trials came to a halt within days of the new U.S. government administration.