Medical records of Sanfilippo children can accelerate discovery of a treatment

September 24, 2020
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Parents and caregivers of children with Sanfilippo Syndrome have the opportunity to help accelerate drug development for the disease right from their homes.

AllStripes, formerly RDMD, is a research platform dedicated to rare diseases that enables patients to contribute to new treatment studies from home. AllStripes does the work to collect and analyze patients’ de-identified medical records to help power faster, better drug development for rare conditions.

Sanfilippo Syndrome is among the rare diseases on which AllStripes is collecting data.

“Compiling data through AllStripes, formerly RDMD, is so very important because it can help many groups (biotech industry, institutions, researchers, FDA, nonprofits) understand what is happening during the life cycle of these children and potentially identify key data points that could be included in trial design (new and ongoing) for quickening the path to a hopeful FDA approval,” said Cure Sanfilippo Foundation Chief Science Officer Cara O’Neill, MD, FAAP.

Participating is as simple as logging into the Sanfilippo-specific section of AllStripes’ website, registering, providing some information about your child’s doctors through the years, and they do the rest. You then have access to all of your child’s medical records all in one place, as a bonus.

Families that have already completed the process say its easy and takes roughly 30 minutes. They also find that it’s a great resources personally when schools request medical records because everything is conveniently located in one place.

Families of children who have passed away from Sanfilippo Syndrome are highly encouraged to participate. “Their medical records and data is incredibly helpful as well,” said O’Neill.

Currently, the records of 37 patients with Sanfilippo have been submitted. The goal is to have 75 in order to have a robust body of data that meaningfully benefits research.

Cure Sanfilippo Foundation is proud to partner with AllStripes on this critical research endeavor and invites families from around the world, representing all types of Sanfilippo, to participate.

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