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 ...

A Phase III study examining a treatment for the rare genetic disorder Niemann-Pick disease has seen 87% of its participants, all younger than three years old, show signs of stabilisation or ...

Niemann-pick type C (NPC) is a rare neurological disorder where harmful levels of lipids (fats) accumulate in the body. Type A is the most serious form of the disorder, with infants succumbing to ...

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 ...

NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9 ... Antibodies to AAV2 and AAV9 in Individuals with ...

Niemann-Pick Disease Market ... This rare lysosomal storage disorder, characterized by the accumulation of sphingomyelin and cholesterol in cells, presents significant challenges, but also ...

This recommendation is not intended to affect treatment with olipudase alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may ...

Zevra Therapeutics publishes key findings on arimoclomol's mechanism for treating Niemann-Pick disease type C in a scientific journal. Zevra Therapeutics, Inc. announced the publication of a study ...

Epilepsy, which is a feature of Niemann–Pick disease type C ... Zebra has another approved and marketed asset, the urea cycle disorder drug Olpruva, which has a small revenue stream.

The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to ...