Motor neuron synapses with muscle fiber via electrical impulse transmission and neurotransmitter release, forming neuromuscular junctions , motor neuron, neuroscience

SOD1-ALS from Gene Discovery to Targeted Therapeutics: A Comprehensive Review

Joshua Newman, Phillipe Corcia, Angela Genge

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of upper and lower motor neurons that results in progressive motor impairment. ALS is the most common disease of motor neurons with an annual incidence of approximately 1.7–2.5 per 100,000 people. It is a terminal condition with a typical life expectancy of 2–5 years from symptom onset. […]

touchREVIEWS in Neurology. 2025;21(1):Online ahead of journal publication

Neurometabolic Disease

Niemann-Pick Disease Type C

Published Online: Jul 24th 2015

Dr. Marc Patterson a pediatric neurologist at Mayo Clinic discusses Niemann-Pick disease type C.

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Tahseen Mozaffar: PROPEL Study Results Investigating the use of AT-GAA for the Treatment of Late-onset Pompe Disease

touchNEUROLOGY caught up with Tahseen Mozaffar (University California, Irvine, CA, USA) to discuss the results from the PROPEL study (NCT03729362) investigating the use of AT-GAA for the treatment of late-onset Pompe disease. Questions: Could you tell us a little about late onset Pompe disease and the unmet needs in its treatment? (0:15) What is AT-GAA […]

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SOD1-ALS from Gene Discovery to Targeted Therapeutics: A Comprehensive Review

Learning Activities

Journals

Specialities

SOD1-ALS from Gene Discovery to Targeted Therapeutics: A Comprehensive Review

Niemann-Pick Disease Type C

This Content is for
Members Only

Tahseen Mozaffar: PROPEL Study Results Investigating the use of AT-GAA for the Treatment of Late-onset Pompe Disease

Antonio Federico, EAN 2018 – Effects of migration on the pathophysiology of neurological conditions

Antonio Federico, EAN 2018 – Involvement of the EAN in rare neurological diseases

Günther Deuschl, EAN 2018 – Burden of neurological diseases across Europe (Part 2)

Günther Deuschl, EAN 2018 – Burden of neurological diseases across Europe (Part 1)

Thapanee Somboon, AAN 2018 – Neurology and sleep-disordered breathing/sleep apnea

New Antisense Therapy in Development for Pompe Disease

Pompe Disease

New Treatment Option for Niemann-Pick Type B Disease

New Treatment Option for Niemann-Pick Type B

New Antisense Therapy in Development for Pompe Disease

Anti-NMDA Receptor Encephalitis- the clinical criteria of early diagnosis

Niemann-Pick type C disease

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