Popular Stories

Takeda Tag

  • All
  • Biotechnology
  • Cannabis
  • Education
  • Investing Ideas
  • Macro
  • Miscellaneous
  • Sectors
  • Startups
  • Technologies
  • Trending
  • Uncategorized
5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

Initiates Make-or-Break Phase 3 Clinical Trials in AngelMan Syndrome

Ovid Therapeutics (NASDAQ: OVID), a biopharmaceutical company developing novel therapeutics for rare neurological disorders, announced the launch of a crucial phase 3 clinical trial in Angelman Syndrome named NEPTUNE. OV101, its lead candidate for  the treatment of syndrome and Fragile X Syndrome is a delta-selective...

WordPress Video Lightbox Plugin