Sinar Daily on MSN7d
Passing On A Destructive Gene
Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a gene ...
BioSpace2dOpinion
Opinion: Companies Vie to Develop a Hunter Syndrome Therapy That Reaches the Brain
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...
Yahoo Finance28d
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II)
for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation ...
Yahoo Finance27d
DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).
Business Insider28d
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II)
About Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals in commercially accessible geographies, primarily males, and leads to physical ...
FierceBiotech22d
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
With a regulatory decision expected before the end of this year, RGX-121 could become the first gene therapy approved for Hunter syndrome. Also known as mucopolysaccharidosis type II, Hunter ...
Investing28d
Denali receives FDA breakthrough status for Hunter syndrome drug
has awarded Breakthrough Therapy Designation to its investigational drug tividenofusp alfa for the treatment of Hunter syndrome, also known as MPS II. This recognition follows previously granted ...