Mucopolysaccharidosis Market Analysis Across 7MM — Unveiling Growth Opportunities Across Different Types | DelveInsight
The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...
Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...
Ultragenyx reports Q4 EPS ($1.39), consensus ($1.27)
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
Nasdaq16d
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).
Central Western Daily16d
'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 lives of diagnosed children and their families.
Investing16d
Ultragenyx reports positive data on gene therapy for rare disease
Notably, older children or those with more advanced disease retained ... received several designations from the FDA that could expedite its review process. Sanfilippo syndrome type A is a rare, fatal ...
Nasdaq17d
Ultragenyx Announces New Data For UX111 Gene Therapy In Treating Sanfilippo Syndrome Type A
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 ...
Investing17d
Ultragenyx reports positive data on gene therapy for rare disease
Notably, older children or those with more advanced ... from the FDA that could expedite its review process. Sanfilippo syndrome type A is a rare, fatal condition with no approved treatment ...
WOOD-TV22d
Charity basketball games to benefit girl with rare genetic disorder
Officers, deputies play basketball for a good cause “She was diagnosed with Sanfilippo Syndrome, which is also called childhood Alzheimer’s. Its neurodegenerative life expectancy is into the ...