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 ...

JCR announced the launch of a film featured in The Next Frontier, a series presented by BIO and produced by BBC StoryWorks ...

Lysosomal Storage Disorders are a rare deadly genetic disorder spreading in India Learn about its causes symptoms ...

Chiesi Group signs global licensing pact with Key2Brain to advance BBB-crossing therapies for lysosomal storage disorders: Parma, Italy Saturday, June 14, 2025, 16:00 Hrs [IST] Ch ...

The Gaucher and Pompe diseases enzyme replacement therapy (ERT) market has seen remarkable evolution over the years. Driven by advancements in biotechnology, increasing awareness of rare genetic ...

Italian family-owned drugmaker Chiesi Group and Sweden’s Key2Brain today announced a worldwide license agreement to advance the development of two blood-brain barrier (BBB)-crossing recombinant enzyme ...

Collaboration will leverage Key2Brain’s technology to develop blood-brain barrier-crossing enzyme replacement therapies for lysosomal storage disorders Agreement is built on a partnership initiated ...