The report also provides a detailed analysis of the current Niemann-Pick disease type C marketed drugs and late-stage pipeline drugs. BROOKLYN, BROOKLYN, UNITED STATES, June 25, 2025 /EINPresswire.com ...

Children with infantile-onset Pompe's disease experienced motor function and cardiac improvements after receiving an ...

"Fabry disease, alpha-mannosidosis, and cystinosis are rare and ultra-rare lysosomal storage disorders causing severe, ...

JCR Pharma introduces mucopolysaccharidosis type II disease awareness film featured in the Next Frontier series: Hyogo, Japan Friday, June 20, 2025, 18:00 Hrs [IST] JCR Pharmaceut ...