According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…
According to a story from globenewswire.com, the biopharmaceutical company Lysogene and the drug developer Sarepta Therapeutics, Inc., have recently announced that the first patient has been dosed in a Phase…
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…
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 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…
Collaboration between the FDNA, the Cure Sanfilippo Foundation, and the Jonah’s Just Begun Foundation has led to technology that successfully recognises the facial phenotype (observable characteristics) of patients with mucopolysaccharidosis…
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…
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…
According to an article from The Huddersfield Daily Examiner, Isla Sykes was diagnosed with mucopolysaccharidosis (MPS III), otherwise known as Sanfilippo syndrome, at age four. Her uncle Ryan Townrow, is…
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