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Giant axonal neuropathy 2

MONDO:0012411

Any giant axonal neuropathy in which the cause of the disease is a mutation in the DCAF8 gene.

Also known as: CMT2 with giant axons, DCAF8 giant axonal neuropathy, HMSN2 with giant axons, autosomal dominant hereditary motor and sensory neuropathy type 2 with giant axons, giant axonal neuropathy caused by mutation in DCAF8, giant axonal neuropathy type 2, GAN2, autosomal dominant Charcot-Marie-Tooth disease type 2 with giant axons

9 clinical trials for this condition and its sub-types.

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Broader categories

Disease (618) Nervous system disorder (217) Hereditary disease (172) Peripheral nervous system disorder (107) Neuromuscular disease (98) Peripheral neuropathy (90) Charcot-Marie-Tooth disease (43) Human disease (14) Hereditary peripheral neuropathy (6) Hereditary neurological disease (5)
Trials to join now! 7 Not yet recruiting 1 Completed 1
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  • Gene therapy readiness check: new study maps virus immunity in muscle disease patients

    Knowledge-focused Not yet recruiting

    This study will test blood samples from 450 people aged 6 to 60 with inherited neuromuscular diseases to see if they have antibodies that could block gene therapy viruses (AAVs). The goal is to understand how common these antibodies are and which patients might be good candidates…

    Phase: NA • Sponsor: Genethon • Aim: Knowledge-focused

    Last updated Jun 27, 2026 13:04 UTC

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