Applying Operation Warp Speed to ultra rare diseases thehill.com/blogs/congress… Rep. Nydia Velazquez FDA Patient Info Can we talk about this? #CUREmpsIIIc

Patient Advocates you can register free now for the annual ASGCT Pre-Meeting workshop, May 10th. I will be speaking the morning of the 10th, title of my talk: 'Gene Therapy for Dummies'. #https://annualmeeting.asgct.org/program/pre-meeting-workshops?_zs=70GHb&_zl=xqPU2

WOooHhhOoo we just broke half a million dollars on our fundraising campaign to raise 3million dollars to fund the manufacturing and trial of a gene therapy treatment. THANK YOU for SUPPORTING our families and giving our kids a chance to FIGHT their FATE! Happy dance.

Calling all RARE disease ADVOCATES who are interested in learning more about gene therapy. You can get a free pass for this years ASGCT pre-meeting workshop. Register here annualmeeting.asgct.org/program/pre-me…

Register now for #ASGCT21! During the premier event in #genetherapy and #celltherapy, you'll find the most educational and scientific programming presented by highly respected researchers in the field. Associate Members can even register for free! bit.ly/3m54mEh

We have big policy goals for 2021, but we need your help! Read about what we hope to accomplish this year and how you can help us advance our goal of unmitigated access to #genetherapy and #celltherapy! bit.ly/3byL08L

Hi friends please vote for our post-doc Masha Taherzadeh, this beautiful image was taken from one of our research programs.#CUREmps NSERC / CRSNG Acfas #ScienceExposed nserc-crsng.gc.ca/ScienceExposed…

For all those patient advocates interested in pursuing gene therapy for your rare disease should register for this conference. Patient Advocates can register for the workshop for $25. asgct.org/.../patient-ad…

Happy Mother's Day all! Loving my new tote that Jonah made me at school. #CUREmpsIIIC

A Mother's Day letter out to billie eilish's mom, #MaggieBared please share. #SaveConnor jonahsjustbegun.blogspot.com

See this Instagram photo by @sanfilippochildrensfoundation instagram.com/p/CO1pKGjDIb-/…

It's National MPS awareness day! In honor of the day please vote for this amazing photo of an MPS IIIC mouse neuron, by Masha Taherzadeh nserc-crsng.gc.ca/ScienceExposed… #ScienceExposed NSERC / CRSNG @MahsaT #CUREmpsIII

2021 Million Dollar Bike Ride- Donation made in memory of Karena Guajardo. Inhttps://support.mpssocietygive.org/MDBR2021 Luis Guajardo #CUREmps National MPS Society

TODAY is CONNOR'S 13th Bday, I'm sure Connor's wish would be along the lines of a new Paw Patrol HQ station. Connor doesn't know he's dying, but his parents do and they want nothing more than to save Connor's life. youtu.be/KlYkzfES-cA via @ColumbusChildrn

Chris Meloni Don't eat my Baby! Eat cake instead! Jonah is turning 13 on 7/30, Connor who is afflicted with MPSIIIC too is turning 13 TODAY! We're raising funds 2 save both our boy's. Sharing is caring. #SaveConnor #SaveJonah gofund.me/bbda3327 @Mariska ICE T BD WONG

Absolutely wonderful! Today is the best day ever and we still have more to come. Looking forward to a cure! #CUREmpsIIIC @saveconnornow instagram.com/p/CQZ-YNin9uC/…

Just posted a photo instagram.com/p/CQg3WrOnpGp/…

Can you help us reach our goal of 1million dollars by June 30th? Our families with children suffering from Sanfilippo type C have great hope that we will see a life saving treatment developed in our children's lifetime! Save Connor Now Saveconnor.com

Such an important cause that gives hope to so many kids and families

It’s been said that Duskull loves the crying of children. It startles bad kids by passing through walls and making them cry. 👻 #PokemonGOCommunityDay