GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on 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).

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.

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 ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behaviorLong ...

Sanfilippo syndrome type A is a rare, fatal condition with no approved treatment that affects approximately 3,000 to 5,000 patients in commercially accessible geographies. Ultragenyx specializes ...

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). Per ...

Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...

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 ...