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What are the latest advances in Sanfilippo Syndrome?

Here you can see the latest advances and discoveries made regarding Sanfilippo Syndrome.

Latest progress of Sanfilippo Syndrome
3 answers
We are researching and conducting clinical trials in ERT and Gene Therapy among others
Posted Jan 1, 2018 by Danielle 3160
Substrate reduction therapies , Enzyme replacement trials underway in 2018 and several gene therapy trials happening in 2019, IV trials for Autophagy underway in 2019 !
Posted Feb 25, 2019 by Patty Jesse 3000
There has been a few clinical trials which sadly haven't been useful in finding a treatment.
Posted Nov 3, 2019 by Anne-marie 2500

Latest progress of Sanfilippo Syndrome

Sanfilippo Syndrome life expectancy

What is the life expectancy of someone with Sanfilippo Syndrome?

6 answers
Celebrities with Sanfilippo Syndrome

Celebrities with Sanfilippo Syndrome

5 answers
Is Sanfilippo Syndrome hereditary?

Is Sanfilippo Syndrome hereditary?

6 answers
Is Sanfilippo Syndrome contagious?

Is Sanfilippo Syndrome contagious?

7 answers
ICD9 and ICD10 codes of Sanfilippo Syndrome

ICD10 code of Sanfilippo Syndrome and ICD9 code

5 answers
Natural treatment of Sanfilippo Syndrome

Is there any natural treatment for Sanfilippo Syndrome?

4 answers
Living with Sanfilippo Syndrome

Living with Sanfilippo Syndrome. How to live with Sanfilippo Syndrome?

4 answers
Sanfilippo Syndrome diet

Sanfilippo Syndrome diet. Is there a diet which improves the quality of lif...

5 answers

World map of Sanfilippo Syndrome

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Stories of Sanfilippo Syndrome

SANFILIPPO SYNDROME STORIES
Sanfilippo Syndrome stories
DIAGNOSING EMILY. A JOURNEY WITH SANFILIPPO SYNDROME
Emily was born a perfectly healthy, 8lb 12oz full term baby girl. She progressed typically until around 3.5yrs old we thought her speech should be a bit more developed than it was. She had also developed what we assumed to be ADHD. Em's first special...
Sanfilippo Syndrome stories
ABBY WALLIS
Abby was thought to have PDD-NOS until October of 2017 when she was diagnosed with Sanfilippo Syndrome-type A. She is 22 years old.
Sanfilippo Syndrome stories
ANTHONY HUDSON
Was 14 years old in 2005 when he gained his wings MPS/sanfilippo/type A
Sanfilippo Syndrome stories
DIAGNOSIS
Reagan was diagnosed at age 3 with MPS III A. Reagan has some hearing loss and speech delay. Reagan is currently involved in a Gene Replacement Therapy Trial.
Sanfilippo Syndrome stories
HEATHER ASHLEY
My daughter was diagnosed with Sanfillippo Syndrome 3 a month after my son passed away at the the age of 26 days old. He was on life support and we has to disconnect life support due to him needing a heart and lung transplant and his organs were fail...

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