Limb-Girdle Muscular Dystrophy — Research Summary
Printed from RareWays (rareways.com.au) on 10 September 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
A bio-inspired spiking neural network with adaptive spatiotemporal filtering and depth-modulated synaptic plasticity for robust collision detection.
Ren Yumeng et al. — Neural networks : the official journal of the International Neural Network Society (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/41855882/
- 2.
Generation of two LGMDR4 patients derived induced pluripotent stem cell line carrying the SGCB frameshift mutation (p.Gly129_Arg130insGlnTer).
Villa Chiara et al. — Stem cell research (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42225003/
- 3.
Expanding the phenotypic spectrum of LAMA2-related disorders: Axonal neuropathy in the absence of muscular dystrophy.
Mohammadi Mahsa et al. — Journal of human genetics (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/41765988/
- 4.
SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.
Iyer Sukanya et al. — Molecular therapy : the journal of the American Society of Gene Therapy (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/41814652/
- 5.
Redox-Encoded Proteostasis: Heat Shock Proteins as Integrators of Stress Adaptation and Therapeutic Targets.
Guo Kangmiao et al. — Antioxidants & redox signaling (1 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42014310/
Clinical Trials — Australian Sites
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
Active (not recruiting) — Phase 3 — ML Bio Solutions, Inc.
https://clinicaltrials.gov/study/NCT07678775
- 2.
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
Active (not recruiting) — Phase 3 — ML Bio Solutions, Inc.
https://clinicaltrials.gov/study/NCT05775848
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.
Limb-Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy encompasses more than 30 distinct genetic subtypes of progressive muscle disease affecting the shoulder and hip girdles. Symptoms typically begin in childhood or early adulthood and include proximal muscle weakness and wasting, with variable rates of progression. Gene therapy trials are active for several subtypes.
Most Recent Research
Achieving fast and robust collision detection on autonomous agents requires perceiving threats and issuing early warnings with minimal latency. Particularly suited for this, dynamic vision sensors (DVS) capture high-speed motion with microsecond temporal resolution, empowering computational models of the lobula giant movement detector (LGMD) to perform rapid and selective collision detection. Aligning with this bio-inspired approach, the neuromorphic architecture and inherent temporal dynamics of spiking neural networks (SNNs) render them an excellent framework for integrating these advantages. However, existing systems still suffer from pronounced noise, highly variable event rates, and limited collision selectivity in complex motion scenarios. To overcome these limitations, we propose an SNN enhancing both robustness and selectivity. Inspired by retinal adaptation, we introduce an adaptive spatiotemporal filtering (ASTF) mechanism. Leveraging spatiotemporal integration and mixed-threshold neurons with global-local adaptation, the ASTF mechanism suppresses hot pixel and background activity (BA) noise and normalizes the output event rate, ensuring stable downstream processing. To further improve looming selectivity, we propose a depth-modulated spike-timing-dependent plasticity (D-STDP) learning rule. This mechanism incorporates a depth-modulated factor derived from global motion cues, which selectively potentiates synapses for looming motion, depresses them for receding motion, and gates off plasticity for irrelevant stimuli. We evaluate the proposed model on a multi-scenario dataset captured with a DVS, containing geometric, ball, and model vehicle motion. The results demonstrate that the proposed model achieves over 94% accuracy across diverse and challenging conditions, providing a promising approach for the development of bio-inspired collision detection visual systems.
Common Questions
What is Limb-Girdle Muscular Dystrophy?
Limb-Girdle Muscular Dystrophy encompasses more than 30 distinct genetic subtypes of progressive muscle disease affecting the shoulder and hip girdles. Symptoms typically begin in childhood or early adulthood and include proximal muscle weakness and wasting, with variable rates of progression. Gene therapy trials are active for several subtypes.
How many clinical trials are available for Limb-Girdle Muscular Dystrophy?
RareWays currently indexes 47 clinical trials for Limb-Girdle Muscular Dystrophy, of which 6 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Limb-Girdle Muscular Dystrophy come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
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