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Sanfilippo Syndrome News Sanfilippo Syndrome News is a digital platform providing daily updates on research, science and advi

Columnist Kelly Wallis writes about how difficult it is to have no control over her daughter's disease — like a plane st...
04/10/2024

Columnist Kelly Wallis writes about how difficult it is to have no control over her daughter's disease — like a plane stuck on the tarmac. https://buff.ly/481mng7

Levels of neurodegeneration-related proteins were elevated in cells derived from Sanfilippo patients relative to cells f...
03/10/2024

Levels of neurodegeneration-related proteins were elevated in cells derived from Sanfilippo patients relative to cells from healthy people. https://buff.ly/4dDdkD7

Columnist Valerie Tharp Byers describes a two-month battle to help her son gain badly needed weight — and the resulting ...
24/09/2024

Columnist Valerie Tharp Byers describes a two-month battle to help her son gain badly needed weight — and the resulting caregiver burnout. https://buff.ly/3TBvmyM

Japan's Pharmaceuticals and Medical Devices Agency cleared a Phase 1 trial of JR-441 for Sanfilippo syndrome type A. htt...
05/09/2024

Japan's Pharmaceuticals and Medical Devices Agency cleared a Phase 1 trial of JR-441 for Sanfilippo syndrome type A. https://buff.ly/47dOiZI

After almost 30 years of advocating for her daughter Abby, who has Sanfilippo syndrome, columnist Kelly Wallis admits sh...
09/08/2024

After almost 30 years of advocating for her daughter Abby, who has Sanfilippo syndrome, columnist Kelly Wallis admits she's tired. https://buff.ly/3YDfEqb

The FDA selected DNL126, Denali Therapeutics' treatment candidate for Sanfilippo syndrome type A, to join its START pilo...
01/08/2024

The FDA selected DNL126, Denali Therapeutics' treatment candidate for Sanfilippo syndrome type A, to join its START pilot program. https://buff.ly/3Sx56Fa

The devastating impact of Hurricane Beryl reminds columnist Valerie Tharp Byers of all her son with Sanfilippo syndrome ...
23/07/2024

The devastating impact of Hurricane Beryl reminds columnist Valerie Tharp Byers of all her son with Sanfilippo syndrome needs. https://buff.ly/3ShKcti

The FDA has granted fast track designation to GC1130A, an enzyme replacement therapy (ERT) being developed for Sanfilipp...
11/07/2024

The FDA has granted fast track designation to GC1130A, an enzyme replacement therapy (ERT) being developed for Sanfilippo syndrome type A. https://buff.ly/3LlEkM4

While celebrating her son's eighth grade graduation, columnist Valerie Tharp Byers can't help but imagine a life without...
25/06/2024

While celebrating her son's eighth grade graduation, columnist Valerie Tharp Byers can't help but imagine a life without Sanfilippo syndrome. https://buff.ly/3VV4Cux

Columnist Emily Wallis remembers the high school days of her and her sister, Abby, who has Sanfilippo syndrome. https://...
17/06/2024

Columnist Emily Wallis remembers the high school days of her and her sister, Abby, who has Sanfilippo syndrome. https://buff.ly/3xbxXaH

Columnist Kelly Wallis' daughter Abby was always affectionate growing up, but Sanfilippo seems to be taking that away, K...
07/06/2024

Columnist Kelly Wallis' daughter Abby was always affectionate growing up, but Sanfilippo seems to be taking that away, Kelly writes. https://buff.ly/4aRL66k

GC Biopharma and Novel Pharma plan a Phase 1 clinical trial this year to test GC1130A, an investigational enzyme replace...
06/06/2024

GC Biopharma and Novel Pharma plan a Phase 1 clinical trial this year to test GC1130A, an investigational enzyme replacement therapy. https://buff.ly/3yPm4Yt

Columnist Valerie Tharp Byers didn't think she'd be moving into a new home this month, but it's for the benefit of her s...
21/05/2024

Columnist Valerie Tharp Byers didn't think she'd be moving into a new home this month, but it's for the benefit of her son with Sanfilippo. https://buff.ly/3V652xE

A new study estimates that the economic burden of Sanfilippo syndrome in the U.S. from 2023 to 2043 will exceed more tha...
02/05/2024

A new study estimates that the economic burden of Sanfilippo syndrome in the U.S. from 2023 to 2043 will exceed more than $2 billion in total. https://buff.ly/4a3hliF

Her son's Sanfilippo syndrome makes an accident even scarier for columnist Valerie Tharp Byers when a head injury requir...
23/04/2024

Her son's Sanfilippo syndrome makes an accident even scarier for columnist Valerie Tharp Byers when a head injury requires a trip to the ER. https://buff.ly/3UvT9jl

Columnist Kelly Wallis has been blindsided by several major losses throughout her life, leaving her afraid of it happeni...
05/04/2024

Columnist Kelly Wallis has been blindsided by several major losses throughout her life, leaving her afraid of it happening again. https://buff.ly/3VNviO5

The international Transpher A study enrolled 28 children with Sanfilippo type A, with all receiving a single UX111 dose ...
04/04/2024

The international Transpher A study enrolled 28 children with Sanfilippo type A, with all receiving a single UX111 dose at one of three doses. https://buff.ly/3TFu3xQ

Columnist Valerie Tharp Byers finds solace in her spring break visits to the Easter Bunny with her teenager with Sanfili...
19/03/2024

Columnist Valerie Tharp Byers finds solace in her spring break visits to the Easter Bunny with her teenager with Sanfilippo syndrome. https://buff.ly/492FTHS

Columnist Kelly Wallis has spent years with the emotional toll of painful baggage, including having a daughter with Sanf...
08/03/2024

Columnist Kelly Wallis has spent years with the emotional toll of painful baggage, including having a daughter with Sanfilippo syndrome. https://buff.ly/48ISuQp

Developing biomarkers would support accelerated federal approval for treatments for Sanfilippo syndrome and MPS diseases...
08/03/2024

Developing biomarkers would support accelerated federal approval for treatments for Sanfilippo syndrome and MPS diseases, presenters said. https://buff.ly/49MUqZx

Columnist Valerie Tharp Byers embraces the accessibility of her local area as a way to make memories with her son, who h...
20/02/2024

Columnist Valerie Tharp Byers embraces the accessibility of her local area as a way to make memories with her son, who has Sanfilippo. https://buff.ly/3UHnI7e

Orphan drug status was granted to GC1130A, an investigational brain-penetrant enzyme replacement therapy for Sanfilippo ...
02/02/2024

Orphan drug status was granted to GC1130A, an investigational brain-penetrant enzyme replacement therapy for Sanfilippo syndrome type A. https://buff.ly/42nbNx3

Separation anxiety is understandable for parents of children with Sanfilippo syndrome. Columnist Valerie Tharp Byers sha...
23/01/2024

Separation anxiety is understandable for parents of children with Sanfilippo syndrome. Columnist Valerie Tharp Byers shares her story. https://buff.ly/491wEs0

While a new year often signals new beginnings, it tends to be more complicated for Sanfilippo families, columnist Kelly ...
05/01/2024

While a new year often signals new beginnings, it tends to be more complicated for Sanfilippo families, columnist Kelly Wallis explains. https://buff.ly/3TPSudE

The U.S. FDA has granted orphan drug status to JR-441, an experimental enzyme replacement therapy for Sanfilippo syndrom...
04/01/2024

The U.S. FDA has granted orphan drug status to JR-441, an experimental enzyme replacement therapy for Sanfilippo syndrome type A https://buff.ly/3tDiP41

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AWIR - Learning From Each Other
AWIR - Learning From Each Other

Patient advocate Liza Bernstein explains how #AWindowIntoRare will highlight what we can learn from each other as members of the #raredisease community. Join #BioNews in an online panel discussion + Q&A on #RareDiseaseDay at 3 p.m. EST Feb 28. Register: https://bit.ly/Bionews_RegisterNow

Rare Disease Day - A Window Into Rare
Rare Disease Day - A Window Into Rare

Patient advocate Liza Bernstein gives some insight into what will be discussed during "A Window Into Rare," our #RareDiseaseDay online panel discussion on Feb. 28. Register for this live event here: https://bit.ly/Bionews_RegisterNow #AWindowIntoRare #BioNews

A Window Into Rare - Rare Disease Day
A Window Into Rare - Rare Disease Day

"I have an understanding of what it feels like to feel isolated and erased": Patient advocate Liza Bernstein is hosting "A Window Into Rare," our #RareDiseaseDay panel discussion on Feb. 28. Register for this live event here: https://bit.ly/Bionews_RegisterNow #AWindowIntoRare #BioNews

We Are Rare. We are BioNews.
We Are Rare. We are BioNews.

Sanfilippo Syndrome News is part of the BioNews family, and 60% of us behind the scenes live with a rare condition. This is why we strive to provide you with trustworthy content, meaningful resources and a safe space. #WeAreRare. We are #BioNews. Learn more: https://bionews.com #RareDiseases #SanfilippoSyndromeNews #SanfilippoSyndrome #LivingwithSanfilippoSyndrome #SanfilippoSyndromeCommunity

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