Batten Disease — Research Summary
Printed from RareWays (rareways.com.au) on 25 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Plaque characteristics and clinical outcomes of non-culprit long lesions in patients with acute myocardial infarction.
Cui Lina et al. — International journal of cardiology (1 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42309490/
- 2.
In situ NMR investigation of the native chemical ligation (NCL) of N-terminal cysteines to alginate.
Bučak Gasser David et al. — Carbohydrate polymers (1 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42285655/
- 3.
Retraction: In vivo NCL targeting affects breast cancer aggressiveness through miRNA regulation.
Pichiorri Flavia et al. — The Journal of experimental medicine (3 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42377339/
- 4.
CLN5 disease-causing mutations impact lysosomal biology by affecting intracellular degradation and protein trafficking.
Kim William D et al. — Biochimica et biophysica acta. Molecular basis of disease (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42031177/
- 5.
Amine-Enabled Electron Donor-Acceptor Complex Catalysis for Cyclopropanation.
Tarkase Rahul et al. — Organic letters (2 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42394196/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting — Sanford Health
https://clinicaltrials.gov/study/NCT01793168
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.
Batten Disease
Batten disease (neuronal ceroid lipofuscinosis) is a group of rare and fatal genetic neurological disorders that cause progressive deterioration of vision, seizures, and cognitive and motor decline, often beginning in childhood. The first approved enzyme replacement therapy (cerliponase alfa) targets CLN2 disease and has slowed progression.
Most Recent Research
BACKGROUND: This study aimed to investigate plaque characteristics and long-term outcomes associated with long non-culprit lesions (NCLs) in patients with acute myocardial infarction (AMI). METHODS: A total of 1278 AMI patients undergoing three-vessel optical coherence tomography (OCT) were retrospectively enrolled, and 5131 NCLs were identified. A long lesion was defined as an OCT lesion ≥20 mm in length. Patients were followed for up to 5 years, and NCL-related major adverse cardiovascular events (NCL-MACE) were recorded. RESULTS: Both at the patient and lesion level, long NCLs were more stenotic and had more frequent thin-cap fibroatheroma (TCFA) and other vulnerable plaque features than short NCLs (all P < 0.001). During a median follow-up of 4.1 years, patients with ≥1 long NCL had a significantly higher incidence of NCL-MACE than patients without long NCL (7.3% vs. 2.9%, adjusted HR: 2.26, 95%CI: 1.19-4.29). Similar findings were identified when patients were grouped by angiographic lesion length. In the lesion-level analysis, OCT-detected long NCLs remained significantly associated with NCL-MACE after adjustment for TCFA (adjusted HR: 1.97, 95%CI: 1.11-3.52), whereas angiography-detected long NCLs showed no prognostic value. Notably, OCT-detected long TCFA had highest lesion-specific risk (5.4% vs. 1.1%, adjusted HR: 3.69, 95%CI: 1.87-7.27), whereas risk of OCT-detected short TCFA was comparable to that of non-TCFA (1.1% vs. 1.1%, P = 0.986). CONCLUSIONS: Long NCLs were indicative of higher levels of pancoronary plaque vulnerability, irrespective of detection via OCT or angiography. Importantly, OCT-detected long NCLs, especially long TCFA, offered significant predictive value for 5-year adverse events. However, angiography-detected long NCLs lacked prognostic significance.
Common Questions
What is Batten Disease?
Batten disease (neuronal ceroid lipofuscinosis) is a group of rare and fatal genetic neurological disorders that cause progressive deterioration of vision, seizures, and cognitive and motor decline, often beginning in childhood. The first approved enzyme replacement therapy (cerliponase alfa) targets CLN2 disease and has slowed progression.
How many clinical trials are available for Batten Disease?
RareWays currently indexes 40 clinical trials for Batten Disease, of which 10 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Batten Disease 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.
For guidance specific to your situation, please speak with your healthcare team.