TMCnet11h
Denali Therapeutics Announces Primary Analysis and Long-Term Follow-Up of Phase 1/2 Study in Hunter Syndrome (MPS II) with Tividenofusp Alfa
Long-term safety data with median follow-up of two years, and out to more than four years, demonstrate that tividenofusp alfa was generally well tolerated Regulatory submission for accelerated ...
FierceBiotech12h
Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...
RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
Ultragenyx announces new safety and efficacy data from its pivotal study of its investigational candidate, UX111, for ...
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 ...
The Pharma Letter16h
Ultragenyx updates on UX111 AAV gene therapy
California, USA-based rare disease company Ultragenyx Pharmaceutical yesterday released new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically-significant ...
'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 ...
Ultragenyx reports positive data on gene therapy for rare disease
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 ...
American Association of Individual Investors2d
Which Is a Better Investment, Ultragenyx Pharmaceutical Inc. or Vericel Corporation Stock?
Learn more about whether Ultragenyx Pharmaceutical Inc. or Vericel Corporation is a better investment based on AAII's A+ ...
Hosted on MSN6d
Charity game to benefit Michigan girl with rare genetic disorder
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 ...
MLive6d
Annual Kozminski basketball tournament to benefit Kent County deputy’s daughter
GRAND RAPIDS, MI -- The annual Robert Kozminski Memorial Basketball Game is Saturday, Feb. 1 and will benefit the young ...
wwmt6d
Annual Kozminski Memorial game honors fallen officer while aiding family in need
The Grand Rapids Police Department will face the Kentwood Police Department in just one of the matchups scheduled for the ...
eLife7d
Molecular Feature-Based Classification of Retroperitoneal Liposarcoma: A Prospective Cohort Study
Department of Gastroenterology, Beijing Friendship Hospital, Capital Medical University, State Key Laboratory for Digestive Health, National Clinical Research Center for Digestive Disease, Beijing ...