Paediatric Services In Netherlands

1 services found

In Netherlands Available In Netherlands Near Netherlands

Oral Treatment for Lysosomal Storage Disorders

by:Azafaros based inNaarden, NETHERLANDS

Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique dual-mode action, making it a promising candidate for modifying diseases irrespective of patient genotypes. Notably effective against conditions such as GM1 and GM2 ...

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