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Fetal therapy offers hope for unborn patients suffering from the rare genetic disease Hunter Syndrome
Before Ryann was born, a prenatal screening revealed he had a genetic disorder known as Hunter Syndrome or Mucopolysaccharidosis Type II. It is a rare genetic disorder that Queen's little brother ...
Denali: Despite ALS Primary Endpoint Failure, Possible Path Forward Remains
Denali Therapeutics Inc.'s stock remains stable despite trial setback in ALS. BLA filing for MPS II treatment on track. Click ...
University of Manchester3mon
Groundbreaking gene therapy trial for Hunter syndrome opens
Five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS ... Children with Hunter syndrome have a missing gene, meaning they cannot produce an important ...
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DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation
Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, ...
University of Manchester4y
University announces partnership with AVROBIO for Hunter syndrome gene therapy
The significant partnership agreement is for the clinical development of an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly ...
pharmaphorum7y
Shire's Hunter Syndrome cognitive impairment drug fails
SHP609 was supposed to correct the deficiency in the iduronate-2-sulfatase enzyme in the CNS and prevent the cognitive decline seen in some Hunter Syndrome cases. Sadly, Shire said the phase 2/3 ...
Yahoo Finance2y
Denali Therapeutics Announces Presentations on DNL310 (ETV:IDS) Development Program in MPS II (Hunter Syndrome) at the Upcoming WORLDSymposium™
About DNL310 and Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disease caused by mutations in the gene that encodes for the enzyme iduronate-2 ...
Denali Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II)
Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment ...
조선일보1mon
GC Green Cross secures Russian approval for Hunterase ICV, treatment for Hunter syndrome
GC Green Cross announced on the 11th that its severe Hunter syndrome treatment ‘Hunterase ICV’ has received product approval from the Russian Federal Ministry of Health. This is the first ...