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+ ...

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

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

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

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

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

We assumed that poor sampling or shipment may have contributed to the false positive result because other enzymes, such as acid β-glucocerebrosidase for Gaucher, acid α-galactosidase A for Fabry, and ...

The Indian government has decided to exempt customs duty on 36 life-saving drugs to enhance access to essential treatments ...