SF4K

The official account for the Sanfilippo Foundaton for Children

[Rare Disease Report @RareDR]

Its international Pompe Disease Day ... Here is John Crowley talking about Extraordinary Measures http://t.co/fnPXwlo82b

[MbelZa @BelenZafra]

Center for Gene Therapy Clinical Trials http://t.co/bv8JKwce02 vía @nationwidekids

[SF4K @sf4k]

@FierceBiotech @JohnCFierce the patients believe in Emil Kakkis. His love for patients drives him to succeed. $RARE

[RareConnect @RareConnect]

@sf4k New online community for #Sanfilippo Children available in 5 languages. https://t.co/xpDkRa1D0r

[Roy Zeighami @zeighami]

I did a fresh update to the sf4k website. http://t.co/UR2RuY6ROC. Check it out!

[Ethan Perlstein 1-to-N @eperlste]

At current snail's pace of orphan drug discovery, it'll take another 10 lifetimes before we have a treatment for all 7,000 #rarediseases.

[AdamNelson @AdamMcNelson]

RT: Help terminally-ill kids by shopping at http://t.co/FgxoOP29Xs and selecting 'Team Sanfilippo'. 0.5% of proceeds go to @teamsanfilippo

[SF4K @sf4k]

Check out @caresync. Retrieves & stores heath records for your family. Effective, painless and affordable! http://t.co/h7LmLwBGK7

[EveryLife Foundation @EveryLifeOrg]

Emil Kakkis: Ultragenyx Announces Collaboration With Kyowa Hakko Kirin to Develop and... http://t.co/Ognl0UsiAW

[FDA Law Blog @fdalawblog]

#FDA Issues Guidance on Expanded Access to Investigational Drugs for Treatment Use — http://t.co/TcAzHQQpeG

[SF4K @sf4k]

AAV9 systemic gene therapy at @nationwidekids receives $3.5M NIH grant #curesanfilippo http://t.co/tWxuvYBMx5

[RDLA @RareAdvocates]

The Conference and Lobby Day are free and open to the public. Go to http://t.co/94QyeMVM to... http://t.co/mmiwQuRl

[Columbus Marathon @CbusMarathon]

Big news! Our board was so INSPIRED by you & the kids that they contributed $150K to @nationwidekids, bringing the total raised to $925K!

[FluGov @FluGov]

Got the #flu? Don’t share it. Stay home for at least 24 hours after your fever goes away. Visit http://t.co/i9ibYqch

[RDLA @RareAdvocates]

Thank you @RepCliffStearns & @EdTowns for your Dedication & Leadership and Work to be a #RAREVoice for the #RAREDisease community!

[SF4K @sf4k]

Speaking tomorrow at the rare caucus briefing hope u and JP can come @michaelcburgess http://t.co/hwP7dA9R

[RDLA @RareAdvocates]

A pledge to tell the FDA that I want Neurodegenerative Lysosomal Storage Diseases to be 1 of the 20. http://t.co/8Jl3p8Uj

[Fight To Live Org @FightToLiveOrg]

Inexcusable that patients have to travel overseas 4 treatment they need." If u agree, support @BilbrayCA50 HR 628 http://t.co/jhBS4Bml