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…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
Have you ever heard of a natural history study? In short, a natural history study studies people who have, or are at risk of developing, a specific medical condition;…
Seven years ago, David and Amber Negele married. The pair couldn’t wait to begin their blissful wedded life together. Just a few years after their marriage, David and Amber decided…
Both the 3rd Annual Gene Therapy for Neurological Disorders meeting and the ADVANCE 2022 Sanfilippo Community Conference were held in July 2022. During both meetings, researchers presented new data from…
When Sophia Scott was growing up, her parents noticed that she was displaying a variety of different potentially concerning traits. They began undergoing some tests. Eventually, just one day before…
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…
In 2019, Connor Dobbyn, now 13, was diagnosed with Sanfilippo syndrome type C, also known as mucopolysaccharidosis III (MPS III). After his diagnosis, his parents came to recognize that there…
According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…
According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…
According to a story from finanznachrichten.de, the biopharmaceutical company Abeona Therapeutics, Inc. recently announced that its experimental medical product ABO-101 has earned Fast Track designation from the US Food and…
REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a…
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 Newsweek, when 25 year old Rebecca Griffiths first knew that her son Reggie, now aged two, was diagnosed with Sanfilippo syndrome, she had to face…
A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…
Terrence Merrill shares the challenging emotional realities of raising a child with Sanfilippo syndrome. You can read more background about the Merrill's battle against this rare disease in our article here.…
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