June 23, 2020June 23, 2020
How Are Different Types of Sanfilippo Syndrome Inherited?
Health Insights
Sanfilippo syndrome refers to a group of disorders caused by mutations in genes that provide instructions for making proteins ... Read more
June 22, 2020June 22, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
cystic fibrosis, News, syndicated
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during ... Read more
June 18, 2020June 18, 2020
Lysogene CEO Karen Aiach, Inspired by Daughter, Determined to Treat Sanfilippo
News
Karen Aiach never imagined she’d one day start a drug company dedicated to curing a fatal genetic disease. But ... Read more
June 17, 2020June 16, 2020
Feeling Guilty About Moving Away as a Sanfilippo Sibling
Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis
I recently accepted a teaching position out of town, and am moving into a new apartment this week. As ... Read more
June 16, 2020June 16, 2020
Reproductive Options for Sanfilippo Syndrome Carriers
Health Insights
The prevalence of Sanfilippo syndrome in the general population is about 1 in 70,000 people. However, if you are a ... Read more
June 12, 2020June 10, 2020
When Pushing Forward Is the Only Direction to Go
Columns, Parenting a Sanfilippo Child – a Column by Kelly Wallis
Loss is a theme that persistently has occupied my thoughts lately, not only because of current events in the ... Read more
June 11, 2020June 11, 2020
FDA Puts Hold on LYS-SAF302 Gene Therapy Trial for Sanfilippo Type A
News
The U.S. Food and Drug Administration (FDA) has issued a hold on Lysogene‘s trial of its gene therapy candidate ... Read more
June 10, 2020June 10, 2020
Coalition Will Address Racial Disparities in Rare Disease Communities
News
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare ... Read more
June 9, 2020June 9, 2020
Sanfilippo Syndrome Type A Versus Type B
Health Insights
Sanfilippo syndrome (also called MPS III) is a rare genetic disease characterized by neurodegeneration, or the death of nerve ... Read more
June 4, 2020June 4, 2020
Seelos Seeks Natural History Data, Rare Disease and Orphan Drug Designations for SLS-005
News
Seelos Therapeutics and the U.S. Food and Drug Administration (FDA) are continuing to discuss the use of Sanfilippo syndrome ... Read more
June 3, 2020June 1, 2020
Life Never Stops with Sanfilippo Syndrome
Columns, Finding My Peace with Sanfilippo – a Column by Emily Wallis
The current state of the world is difficult for everyone. I’ve thought a lot about my sister, Abby, my ... Read more
June 2, 2020June 2, 2020
Genetic Counseling with Sanfilippo Syndrome and Why It’s Important
Health Insights
Genetic counseling helps you understand how a genetic disease can affect you and your family, and your risk of ... Read more

June 23, 2020June 23, 2020

How Are Different Types of Sanfilippo Syndrome Inherited?

Health Insights

Sanfilippo syndrome refers to a group of disorders caused by mutations in genes that provide instructions for making proteins that play a role in the pathways for breaking down ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

cystic fibrosis, News, syndicated

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 18, 2020June 18, 2020

Lysogene CEO Karen Aiach, Inspired by Daughter, Determined to Treat Sanfilippo

News

Karen Aiach never imagined she’d one day start a drug company dedicated to curing a fatal genetic disease. But that was before 2005, when the French business consultant gave ... Read more

June 17, 2020June 16, 2020

Feeling Guilty About Moving Away as a Sanfilippo Sibling

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

I recently accepted a teaching position out of town, and am moving into a new apartment this week. As exciting as this is, I can’t help but be sad ... Read more

June 16, 2020June 16, 2020

Reproductive Options for Sanfilippo Syndrome Carriers

Health Insights

The prevalence of Sanfilippo syndrome in the general population is about 1 in 70,000 people. However, if you are a carrier of the disease, the risk of passing the rare metabolic ... Read more

June 12, 2020June 10, 2020

When Pushing Forward Is the Only Direction to Go

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

Loss is a theme that persistently has occupied my thoughts lately, not only because of current events in the world, but also because of my personal life. At 24, ... Read more

June 11, 2020June 11, 2020

FDA Puts Hold on LYS-SAF302 Gene Therapy Trial for Sanfilippo Type A

News

The U.S. Food and Drug Administration (FDA) has issued a hold on Lysogene‘s trial of its gene therapy candidate LYS-SAF302 for Sanfilippo syndrome type A. LYS-SAF302 is a one-time ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a ... Read more

June 9, 2020June 9, 2020

Sanfilippo Syndrome Type A Versus Type B

Health Insights

Sanfilippo syndrome (also called MPS III) is a rare genetic disease characterized by neurodegeneration, or the death of nerve cells. There are four subtypes of Sanfilippo syndrome, marked as ... Read more

June 4, 2020June 4, 2020

Seelos Seeks Natural History Data, Rare Disease and Orphan Drug Designations for SLS-005

News

Seelos Therapeutics and the U.S. Food and Drug Administration (FDA) are continuing to discuss the use of Sanfilippo syndrome natural history data as a control for a pivotal U.S. ... Read more