Cyclo Therapeutics’ Trappsol Cyclo was able to induce clinical improvement in seven out of eight children with Neimann-Pick ...

As with the other types of Niemann-Pick disease, this is an autosomal recessive disorder which means that a child or adult with the disease must inherit the genes from both parents (who typically ...

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients. IntraBio's ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease. The US regulator has cleared Miplyffa (arimoclomol ...

What is Niemann-Pick disease type C? Niemann-Pick disease type C is a rare neurodegenerative disorder caused by cholesterol transport issues, leading to severe neurological impairments.

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