Motor Neuron Disease — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Exosome-like nanovesicles from acerola for CRISPR-Cas9 ribonucleoprotein delivery to the central nervous system.
Nagamatsu Yui et al. — Molecular therapy. Nucleic acids (16 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41909467/
- 2.
Differential quadruple pattern: A new EEG signal classification framework.
Ozgor Bilge et al. — IBRO neuroscience reports (1 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41907560/
- 3.
Hereditary transthyretin amyloidosis mimicking ALS: First genetically proven case report from Saudi Arabia.
Elshony Hosna et al. — eNeurologicalSci (1 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41907197/
- 4.
PatientFlow: Learning to generate mixed-type longitudinal clinical data with flow matching.
Branco Ruben et al. — Artificial intelligence in medicine (1 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41830733/
- 5.
Comment on: "Clinical safety of ultra-high-dose methylcobalamin in patients with amyotrophic lateral sclerosis: Open-label extension of a phase 2/3 randomized controlled study".
Wang Rui et al. — Journal of the neurological sciences (15 May 2026)
https://pubmed.ncbi.nlm.nih.gov/41856848/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
Recruiting — Phase 1 — Regeneron Pharmaceuticals
https://clinicaltrials.gov/study/NCT06351592
- 2.
Platform Trial to Assess the Efficacy of Multiple Drugs in Amyotrophic Lateral Sclerosis (ALS)
Recruiting — Phase 3 — Stichting TRICALS Foundation
https://clinicaltrials.gov/study/NCT06008249
- 3.
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Recruiting — Phase 3 — Novartis Pharmaceuticals
https://clinicaltrials.gov/study/NCT05335876
- 4.
Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease
Recruiting — Na — University of Melbourne
https://clinicaltrials.gov/study/NCT05136222
- 5.
Trial of Enhanced Neurostimulation for Anorexia
Recruiting — Na — The George Institute
https://clinicaltrials.gov/study/NCT05788042
- 6.
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
Recruiting — Phase 3 — Celgene
https://clinicaltrials.gov/study/NCT06764485
- 7.
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)
Recruiting — Phase 1 — Novartis Pharmaceuticals
https://clinicaltrials.gov/study/NCT07190300
- 8.
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Recruiting — Phase 1 — Passage Bio, Inc.
https://clinicaltrials.gov/study/NCT04747431
- 9.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting — Sanford Health
https://clinicaltrials.gov/study/NCT01793168
- 10.
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
Recruiting — Phase 1 — Celosia Therapeutics Pty Ltd
https://clinicaltrials.gov/study/NCT07401121
- 11.
Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS
Recruiting — Phase 2 — Mario Negri Institute for Pharmacological Research
https://clinicaltrials.gov/study/NCT06126315
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Motor Neuron Disease
Motor neuron disease (MND), also known as ALS, is a progressive neurodegenerative condition that destroys the nerve cells controlling movement, leading to muscle weakness, paralysis, and eventually respiratory failure. Most cases are fatal within 2-5 years of diagnosis. Riluzole and edaravone are approved treatments; antisense oligonucleotide therapies targeting SOD1 and FUS are transforming outcomes for genetic subtypes.
Most Recent Research
An aberrant six-base repeat in intron 1 of C9orf72 is the most frequent cause of solitary and familial amyotrophic lateral sclerosis and frontotemporal dementia. This mutation is a potential target for CRISPR/Cas9-based genome editing. However, the blood-brain barrier and limitations of current viral or nanoparticle-based delivery systems to neurons significantly restrict the clinical application of CRISPR-Cas9 in the brain. To address these challenges, we developed a drug delivery system using acerola-derived exosome-like nanoparticles (AELNs), which may overcome several limitations associated with human exosomes. AELNs stably form complexes with ribonucleoproteins (RNPs) comprised of Cas9 proteins and guide RNAs (gRNAs). We improved the delivery efficiency and selectivity of AELN/RNP complexes in GLP2-receptor-expressing neurons by incorporating GLP2 peptides into the AELN/RNP complexes. Intranasal administration of peptide-tagged AELN/RNP complexes in vivo confirmed the successful genome editing of C9orf72, demonstrating the potential of this system for treating neurodegenerative diseases. This study presents a potentially innovative approach for in vivo genome editing using a noninvasive delivery system.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.