Posts by: Emily Malcolm, PhD

Emily Malcolm, PhD

Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.

May 26, 2020May 27, 2020

Challenges of Enzyme Replacement Therapy for Sanfilippo Syndrome

Health Insights

Sanfilippo syndrome is a lysosomal storage disorder. Mutations in one or more of four genes — GNS, HGSNAT, NAGLU, and SGSH — can cause the disease. These four genes are important for the function of ... Read more

May 10, 2020May 11, 2020

Clinical Trials and Sanfilippo Syndrome

Health Insights

Patients with rare diseases like Sanfilippo syndrome can help with research into treatments for the disease by participating in clinical trials. They may also have access to new treatments being ... Read more

May 4, 2020May 5, 2020

How Does Sanfilippo Syndrome Differ From Other MPS Types?

Health Insights

Sanfilippo syndrome is a type of mucopolysaccharidosis (MPS), which are lysosomal storage disorders. The lysosome is a compartment within cells that functions as the “garbage collectors,” collecting cellular waste ... Read more

April 27, 2020April 28, 2020

Carpal Tunnel Syndrome in Children With Sanfilippo Syndrome

Health Insights

Sanfilippo syndrome is a rare progressive disease that affects the nerve cells. The disease can cause a condition known as carpal tunnel syndrome, among other symptoms. What is carpal ... 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 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

February 27, 2020February 28, 2020

Importance of Support Networks for Families Affected by Sanfilippo Syndrome

Health Insights

Learning that your child or other family member has a rare disease like Sanfilippo syndrome can bring a tremendous amount of pressure to bear. It’s not uncommon for parents ... Read more

February 13, 2020February 14, 2020

Support Groups for Families Affected by Sanfilippo Syndrome

Health Insights

Families affected by a rare disease such as Sanfilippo syndrome may struggle with stress, depression, and feelings of isolation. If you have a family member with the disorder, it ... Read more

February 6, 2020February 7, 2020

Travel Tips for Families Affected by Sanfilippo Syndrome

Health Insights

Traveling can be challenging for people with Sanfilippo syndrome, depending on the severity of symptoms and the nature of travel. Here are some tips to help you travel safely ... Read more

January 23, 2020January 24, 2020

Music Therapy for People with Sanfilippo Syndrome

Health Insights

Many with Sanfilippo syndrome are non-verbal, and have difficulty communicating with their parents or caregivers. Music therapy may be used to help these patients with their verbal, motor, and social ... Read more

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