Mary Chapman, features writer —

Developing biomarkers may help support accelerated federal approval for treatments in Sanfilippo syndrome and related diseases. That was the subject of an in-person and virtual workshop last month by the Reagan-Udall Foundation for the Food and Drug Administration that discussed primary disease activity biomarkers and focused on…

A primary goal of this year’s World Sanfilippo Awareness Day, observed annually on Nov. 16, is to get people talking about Sanfilippo syndrome, a rare disorder thought to affect 1 in 70,000 people. “World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo syndrome,…

The National MPS Society will host its Maritime Gala May 13 on the San Francisco Bay in Tiburon, California, at the historic Corinthian Yacht Club to raise funds for its family support and research programs. The event also observes International MPS Awareness Day, observed annually on May…

From joining the “Hands of Hope” challenge to adding a Facebook profile frame, supporters globally are poised to mark World Sanfilippo Awareness Day, observed each Nov. 16 to bring attention to the genetic disorder thought to affect one in 70,000 individuals. The awareness campaign targets the general public…

In March, after Alina and Jeremy Croke shared a TikTok video that communicated their $1 million goal to help find a treatment for their young son, Simon, and others who have Sanfilippo syndrome, they were taken aback by the support. The video went viral. What truly surprised the…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

A film crew visited Alina and Jeremy Croke’s Texas home last spring to make a video about their young son, Simon, in hopes of raising $1 million to help find a treatment for Sanfilippo syndrome type B. That video, his parents hoped, might help to save 5-year-old Simon’s life.

When 11-year-old Connor Ridley heard about another boy named Connor who wants to get into a potentially life-changing clinical trial, he decided to help. The other boy, Connor Dobbyn, is 13 and was diagnosed in 2019 with Sanfilippo syndrome, a neurodegenerative disease that renders the body unable to break…

From posting a photo of their child’s hand in theirs, to switching out their Facebook frame, supporters are poised to mark World Sanfilippo Awareness Day, observed each Nov. 16. The aim is to heighten awareness of Sanfilippo syndrome, a genetic disorder that leads to a form of childhood dementia,…

As part of its Innovative Research Grants Initiative, the National MPS Society has awarded $350,000 to support five projects into potential ways of better treating mucolipidoses (ML) or mucopolysaccharidosis (MPS) diseases, such as Sanfilippo syndrome (MPS III). These awards are were given as “cycle 1” of the grant initiative, according…