Mary Chapman, features writer —

Because of Sanfilippo syndrome‘s progressive behavioral and intellectual disabilities, it’s likely your child will need aids and adaptations to help with everyday life. The following information may be useful as you consider those options. About Sanfilippo syndrome Sanfilippo syndrome typically progresses in three stages, beginning with developmental…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

If your child has Sanfilippo syndrome, physiotherapy can likely help with symptoms that include spasticity and the progressive loss of mobility. Here are some things to consider. What is Sanfilippo syndrome? Sanfilippo syndrome is a rare neurodegenerative disorder characterized by continuous intellectual and behavioral disability. Typically,…

Sanfilippo syndrome is a rare neurodegenerative disorder that causes intellectual and behavioral disabilities. Sleep problems are common, and often severe and difficult to manage. Here are some points to consider, including possible ways of managing disturbed sleep. What causes Sanfilippo syndrome? Mutations in genes that provide instructions for…

Providing constant care and supervision for a child with Sanfilippo syndrome can be an emotionally draining task for parents. Beyond handling physical challenges and medical demands, you must deal with your child’s emotional needs and the impact a protracted illness can have on your family. A practice…

Receiving a Sanfilippo syndrome diagnosis for your child may have come as a shock. But now that you know, you need to plan for the future. What causes Sanfilippo syndrome? Sanfilippo syndrome refers to a group of disorders caused by mutations in genes that provide instructions for…

Healthcare technology company RDMD has raised $14 million to further expand its platform seeking to accelerate research and treatment development for rare diseases, including Sanfilippo syndrome. The Series A financing was led by Spark Capital, with participation from Lux Capital, Village Global,…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Axovant Gene Therapies is collaborating with Invitae to offer free genetic testing in the United States and Canada to children suspected of having a lysosomal storage disease. The Axovant sponsorship is meant to help bring down barriers to genetic diagnoses and counseling for lysosomal storage diseases…