Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...

NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating ...

An Orange couple who lost their son to a rare genetic condition are determined to continue raising awareness to improve the ...

The Grand Rapids Police Department will face the Kentwood Police Department in just one of the matchups scheduled for the ...

The daughter of a Kent County sheriff’s deputy who is fighting a rare genetic disorder will benefit from this year’s Robert ...

GRAND RAPIDS, MI -- The annual Robert Kozminski Memorial Basketball Game is Saturday, Feb. 1 and will benefit the young ...

The daughter of a Michigan sheriff’s deputy who is fighting a rare genetic disorder will benefit from the department’s annual charity event called the Robert Kozminski Memorial Basketball Games. The ...

Learn more about whether Ultragenyx Pharmaceutical Inc. or Vericel Corporation is a better investment based on AAII's A+ ...

The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Denali’s candidate is its DNL12 enzyme replacement therapy (ERT) for mucopolysaccharidosis (MPS) IIIA, a form of lysosomal storage disease also known as Sanfilippo syndrome type A, which is in a ...

Carolina DeBenedittis Sanfilippo, 88 of Endicott, NY, passed away on Tuesday January 28, 2025. Carolina was born on November 4, 1936 in Scranton, PA to Elizabeth and Anthony DeBenedittis.