For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.
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RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug StudyUltragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...
Stifel analysts confirmed their Buy rating on Denali Therapeutics Inc. (NASDAQ:DNLI) with a steady price target of $37.00. According to InvestingPro data, analysts maintain a Strong Buy consensus with ...
Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...
An Orange couple who lost their son to a rare genetic condition are determined to continue raising awareness to improve the ...
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, stated, "The data support UX111's potential to provide meaningful benefits to children with Sanfilippo Syndrome, especially for those in ...
Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...
Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).
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