January 17, 2020January 16, 2020
Grieving My Living Daughter
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
My daughter, Abby, is 24 and has Sanfilippo syndrome, which will one day claim her life. I already feel ... Read more
January 16, 2020January 16, 2020
SOBI003 Modified Version of Sulfamidase for Sanfilippo Type A Better at Reaching, Persisting in Brain, Rat Study Suggests
News
SOBI003, Swedish Orphan Biovitrum AB (Sobi)’s chemically modified version of sulfamidase — the missing enzyme in Sanfilippo syndrome type A ... Read more
January 9, 2020January 10, 2020
Two New NAGLU-deficient Stem Cell Lines May Help Study Sanfilippo Type B
News
Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of Sanfilippo ... Read more
January 9, 2020January 10, 2020
How Will Sanfilippo Syndrome Affect My Child’s Life Expectancy?
Health Insights
As a parent, learning that your child has a rare disease like Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS ... Read more
January 8, 2020January 7, 2020
With the New Year Comes Difficult Feelings
Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis
Jan. 1 always brings up difficult feelings for me. Even before Sanfilippo syndrome entered our lives, New Year’s was ... Read more
January 2, 2020January 2, 2020
Lysogene’s Gene Therapy Seen to Correct Enzyme Deficiency in Sanfilippo Type A Mouse Model
News
Lysogene’s gene therapy candidate LYS-SAF302 corrected enzymatic deficiency, normalized heparan sulfate levels, and reduced brain lesions and neuroinflammation in ... Read more
December 20, 2019December 18, 2019
I’m Struggling with Bitterness This Holiday Season
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
Parenting a terminally ill child carries emotional baggage. It can be subtle or sudden and feel like an extra ... Read more
December 19, 2019December 19, 2019
Heparan Sulfate Accumulation May Alter Sulfur Metabolism in Sanfilippo Type B, Mouse Study Suggests
News
The accumulation of heparan sulfate in Sanfilippo syndrome type B can alter sulfur metabolism in the body, which seems ... Read more
December 18, 2019December 16, 2019
Dealing with Jealousy and Other Emotions
Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis
In this column, I want to focus on a word that has been particularly difficult for me to talk ... Read more
December 12, 2019December 12, 2019
Gene Therapy Has Potential to Treat Underlying Cause of Sanfilippo, Review Study Finds
News
Gene therapy has the potential to effectively target the underlying cause of Sanfilippo syndrome and delay or prevent neurodegeneration, according ... Read more
December 11, 2019December 10, 2019
Gene Therapy Takes Center Stage at 2019 NORD Summit
News
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of ... Read more
December 6, 2019December 4, 2019
Dealing with the Emotional Toll of Sanfilippo Syndrome
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
Parents of terminally ill and special-needs children experience a multitude of emotions. Our feelings lie across a wide spectrum, ... Read more

January 17, 2020January 16, 2020

Grieving My Living Daughter

Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis

My daughter, Abby, is 24 and has Sanfilippo syndrome, which will one day claim her life. I already feel like she’s gone, though. I had never even thought of ... Read more

January 16, 2020January 16, 2020

SOBI003 Modified Version of Sulfamidase for Sanfilippo Type A Better at Reaching, Persisting in Brain, Rat Study Suggests

News

SOBI003, Swedish Orphan Biovitrum AB (Sobi)’s chemically modified version of sulfamidase — the missing enzyme in Sanfilippo syndrome type A — is superior to its original version at reaching and ... Read more

January 9, 2020January 10, 2020

Two New NAGLU-deficient Stem Cell Lines May Help Study Sanfilippo Type B

News

Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of Sanfilippo syndrome type B — using CRISPR-Cas9 gene editing technology. Since ... Read more

January 9, 2020January 10, 2020

How Will Sanfilippo Syndrome Affect My Child’s Life Expectancy?

Health Insights

As a parent, learning that your child has a rare disease like Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), can be very difficult. One of the first questions ... Read more

January 8, 2020January 7, 2020

With the New Year Comes Difficult Feelings

Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis

Jan. 1 always brings up difficult feelings for me. Even before Sanfilippo syndrome entered our lives, New Year’s was a holiday for reflection — which isn’t necessarily an easy ... Read more

January 2, 2020January 2, 2020

Lysogene’s Gene Therapy Seen to Correct Enzyme Deficiency in Sanfilippo Type A Mouse Model

News

Lysogene’s gene therapy candidate LYS-SAF302 corrected enzymatic deficiency, normalized heparan sulfate levels, and reduced brain lesions and neuroinflammation in a mouse model of Sanfilippo syndrome type A. LYS-SAF302’s use ... Read more

December 20, 2019December 18, 2019

I’m Struggling with Bitterness This Holiday Season

Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis

Parenting a terminally ill child carries emotional baggage. It can be subtle or sudden and feel like an extra weight to carry around every day. The feeling of bitterness ... Read more

December 19, 2019December 19, 2019

Heparan Sulfate Accumulation May Alter Sulfur Metabolism in Sanfilippo Type B, Mouse Study Suggests

News

The accumulation of heparan sulfate in Sanfilippo syndrome type B can alter sulfur metabolism in the body, which seems to be sex- and organ-specific, a mouse study suggests. The ... Read more

December 18, 2019December 16, 2019

Dealing with Jealousy and Other Emotions

Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis

In this column, I want to focus on a word that has been particularly difficult for me to talk about in the past. While it has synonyms such as ... Read more

December 12, 2019December 12, 2019

Gene Therapy Has Potential to Treat Underlying Cause of Sanfilippo, Review Study Finds

News

Gene therapy has the potential to effectively target the underlying cause of Sanfilippo syndrome and delay or prevent neurodegeneration, according to a review study. Data from ongoing and future clinical ... Read more