After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Through policy framework shifts, appeals to the court for rare disease funds, and starting a centre in Bengaluru, Prasanna ...

Learn more about whether Ultragenyx Pharmaceutical Inc. or Vericel Corporation is a better investment based on AAII's A+ ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will ...

Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...

Dr. Scott Loiler The National MPS Society is pleased to announce the appointment of Dr. Scott Loiler as its new Chief Scientific Officer (C ...

KBC Group NV lifted its holdings in shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report) by 2,275.8% during ...

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

SG Americas Securities LLC trimmed its position in shares of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE – Free Report) by 21 ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present at the 21st Annual WORLDSymposiumtm 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations ...

REGENXBIO Inc., a leading clinical-stage biotechnology company, and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced a strategic partnership for the development and commercialization of RGX-121 ...