manilatimes8h
GC Biopharma Shares Research Updates on Rare Disorder Drugs at the WORLD Symposium 2025
GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on its ...
Onlymyhealth16d
What Is Sanfilippo Syndrome: Symptoms, Treatment, and the Impact on Children’s Lives
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.
Stifel maintains Buy on Denali Therapeutics, $37 target
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 ...
Ultragenyx announces new data on UX111 AV gene therapy
Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...
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Annual Kozminski Basketball Game to benefit deputy’s daughter with rare syndrome
Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...
'Surely there's something we can do': couple's dedication after loss of child
An Orange couple who lost their son to a rare genetic condition are determined to continue raising awareness to improve the ...
Nasdaq4d
RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
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).