FierceBiotech18h
Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...
TheBetterIndia19h
‘No One Should Face This’: After Losing Daughter, Dad Ensures No Rare Disease Patient Suffers Alone
Through policy framework shifts, appeals to the court for rare disease funds, and starting a centre in Bengaluru, Prasanna ...