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 ...

NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9 ... Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1, Human Gene Therapy (2025).

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 ...

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 ...

Within a few months, there was a diagnosis: Niemann-Pick disease type C (NPC). "It was terrifying," said Jessica Martin. NPC is a rare, inherited, progressive and fatal neurodegenerative disease.

The firm focuses on the treatment of rare neurological and sleep conditions such as certain Urea Cycle Disorders, Niemann Pick Disease Type C and Idiopathic Hypersomnia. The company receives ...

The application of HDAC inhibitors for the treatment of various CNS disorders has emerged in ... cholesterol metabolism in models of Niemann–Pick type C disease. A link between metabolism ...

attention deficit hyperactivity disorder (ADHD), Parkinson’s disease, Alzheimer’s disease, Niemann-Pick disease type C, pain disorders, neuromuscular disorders and brain tumours. Members of the ...