March 27, 2020March 25, 2020
The COVID-19 Pandemic Shows How the Medically Fragile Often Live
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
There’s nothing like a global pandemic to provide some perspective. Being forced to stay home, practice social distancing, and ... Read more
March 26, 2020March 26, 2020
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
News
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been ... Read more
March 23, 2020March 23, 2020
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
News
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy ... Read more
March 22, 2020March 23, 2020
Tips for Educators of Children With Sanfilippo Syndrome
Health Insights
Children with special needs can bring certain challenges for caregivers and educators. Young patients with Sanfilippo syndrome face unique ... Read more
March 19, 2020March 19, 2020
New Sanfilippo-like Cell Lines Created from Stem Cells, Study Contends
News
Two types of nerve cells, namely neurons and astrocytes, with Sanfilippo syndrome-like characteristics have been created by converting stem ... Read more
March 13, 2020March 13, 2020
Building an Individualized Education Plan (IEP) for Children with Sanfilippo Syndrome
Health Insights
Sanfilippo syndrome is a rare genetic disorder that causes brain damage and physical and developmental regression in children. These ... Read more
March 13, 2020March 11, 2020
Rallying Support to Raise Awareness and Find a Cure for Sanfilippo Syndrome
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
My daughter Abby has Sanfilippo syndrome, a rare and terminal disease with no cure. Every year my husband, Jeff, ... Read more
March 12, 2020March 12, 2020
Study Examines Newborn Screening for Lysosomal Storage Diseases in Taiwan
News
A multiplex test using a drop of dried blood can be used to screen newborns for eight different lysosomal ... Read more
March 12, 2020March 12, 2020
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
News
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. ... Read more
March 6, 2020March 6, 2020
Early Spinal Surgery May Lessen Nerve Injury in Sanfilippo Syndrome
News
Medical records of children with diseases related to the buildup of glycosaminoglycans, including Sanfilippo syndrome, show that spinal fusion ... Read more
March 5, 2020March 5, 2020
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
News
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients ... Read more
March 4, 2020March 3, 2020
Being a Sanfilippo Sibling Will Make Me a Better Teacher
Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis
Last week, I passed an exam that will allow me to begin teaching this fall. I thought it would ... Read more

March 27, 2020March 25, 2020

The COVID-19 Pandemic Shows How the Medically Fragile Often Live

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

There’s nothing like a global pandemic to provide some perspective. Being forced to stay home, practice social distancing, and work less can alter your point of view. It makes ... Read more

March 26, 2020March 26, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 23, 2020March 23, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 22, 2020March 23, 2020

Tips for Educators of Children With Sanfilippo Syndrome

Health Insights

Children with special needs can bring certain challenges for caregivers and educators. Young patients with Sanfilippo syndrome face unique problems that caregivers should discuss with daycares and schools to ... Read more

March 19, 2020March 19, 2020

New Sanfilippo-like Cell Lines Created from Stem Cells, Study Contends

News

Two types of nerve cells, namely neurons and astrocytes, with Sanfilippo syndrome-like characteristics have been created by converting stem cells with disease-associated mutations, according to a study from researchers ... Read more

March 13, 2020March 13, 2020

Building an Individualized Education Plan (IEP) for Children with Sanfilippo Syndrome

Health Insights

Sanfilippo syndrome is a rare genetic disorder that causes brain damage and physical and developmental regression in children. These symptoms make school and daycare challenging for children with the ... Read more

March 13, 2020March 11, 2020

Rallying Support to Raise Awareness and Find a Cure for Sanfilippo Syndrome

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

My daughter Abby has Sanfilippo syndrome, a rare and terminal disease with no cure. Every year my husband, Jeff, my other daughter Emily, and I host a 5K walk/run ... Read more

March 12, 2020March 12, 2020

Study Examines Newborn Screening for Lysosomal Storage Diseases in Taiwan

News

A multiplex test using a drop of dried blood can be used to screen newborns for eight different lysosomal storage diseases, including Sanfilippo syndrome, a new study shows. The ... Read more

March 12, 2020March 12, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 6, 2020March 6, 2020

Early Spinal Surgery May Lessen Nerve Injury in Sanfilippo Syndrome

News

Medical records of children with diseases related to the buildup of glycosaminoglycans, including Sanfilippo syndrome, show that spinal fusion surgery helped to ease the damage associated with these conditions, ... Read more