Sanfilippo Syndrome: A Doctor and Mother’s Message to the FDA
Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
Fundraising is extremely important when it comes to rare disease research, and recently a community came together to help a little boy living with Sanfilippo syndrome. Simon is 5 years…
Connor Dobyn's parents recognized that their son was missing important developmental delays early on. They brought their concern to the doctor, where Connor was diagnosed with autism. For the next…
According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…
According to a story from metro.co.uk, Harley Bond was three years old when he was first diagnosed with Sanfilippo syndrome type B, a rare progressive genetic disorder. Now five years…
According to an article from R&D, researchers at the New York City-based Icahn School of Medicine at Mount Sinai have entered an agreement with two pharmaceutical companies to aid in…
According to a story from publicnow.com, the drug development company Sarepta Therapeutics recently announced that is has completed negotiations for a licensing agreement with Lysogene, a biopharmaceutical company. This agreement…
Researchers have carried out a literature review to investigate MPS III. After reviewing forty-six papers, they concluded that more research needs to be carried out. You can find the original study…
According to a story from tapinto.net, the Merrill family was crushed upon learning that their five-year-old daughter Leila was born with Sanfilippo syndrome, a lysosomal storage disease that ultimately causes…