Amyloidosis — Research Summary
Printed from RareWays (rareways.com.au) on 26 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Performance and additional benefits of MALDI-TOF-MS in M-protein detection in plasma cell disorders.
Dong Mengmeng et al. — Annals of medicine (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/42046421/
- 2.
Frequency-adjusted daratumumab-based regimen versus bortezomib/dexamethasone in newly diagnosed AL amyloidosis: a matched-cohort study.
Zheng Wanting et al. — Annals of medicine (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/41560662/
- 3.
Tenosynovial and Transverse Carpal Ligament Biopsy for Early Transthyretin Amyloidosis Cardiomyopathy Detection: A Systematic Review and African American Retrospective Cohort Study.
Dogar Shireen et al. — Journal of hand surgery global online (1 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42369086/
- 4.
Cardiac Amyloidosis: Pathogenesis, Diagnosis, and Treatment.
Beckmann Sophie et al. — Deutsches Arzteblatt international (7 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42265992/
- 5.
Association between baseline left ventricular end-systolic diameter and one-year troponin response to tafamidis in transthyretin cardiac amyloidosis.
Morishita Kei et al. — International journal of cardiology. Heart & vasculature (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42291444/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) Amyloidosis
Recruiting — Phase 3 — Novo Nordisk A/S
https://clinicaltrials.gov/study/NCT07207811
- 2.
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Recruiting — Phase 3 — Alnylam Pharmaceuticals
https://clinicaltrials.gov/study/NCT07223203
- 3.
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Recruiting — Phase 3 — Alnylam Pharmaceuticals
https://clinicaltrials.gov/study/NCT07052903
- 4.
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Recruiting — Phase 3 — Intellia Therapeutics
https://clinicaltrials.gov/study/NCT06128629
- 5.
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Recruiting — Phase 3 — Eidos Therapeutics, a BridgeBio company
https://clinicaltrials.gov/study/NCT06563895
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.
Amyloidosis
Amyloidosis is a group of conditions where abnormal proteins called amyloid fibres build up in organs, causing progressive organ damage. ATTR amyloidosis (transthyretin) and AL amyloidosis are the most common types. New RNA-based therapies and small molecule stabilisers have significantly improved outcomes.
Most Recent Research
PURPOSES: Current methods for detecting monoclonal (M) proteins, such as immunofixation electrophoresis (IFE), serum protein electrophoresis (SPEP) and serum free light chains (sFLC), are limited by insufficient sensitivity and suboptimal efficiency. This study evaluated the performance and supplementary value of matrix-assisted laser desorption/ionization-time-of-flight mass spectrometry (MALDI-TOF-MS) for the detection of M-protein. METHODS: The M protein in the blood samples of 137 newly diagnosed plasma cell disorder (PCD) patients were detected using MALDI-TOF-MS. Using SPEP/IFE/sFLC as the gold standard, the performance of MALDI‑TOF MS was assessed; discrepant results were confirmed by urine IFE. RESULTS: The cohort included multiple PCD subtypes, detailly, 116 multiple myeloma (MM, 84.7%), 7 amyloid light-chain (AL) amyloidosis (5.1%), 6 MM combined with AL amyloidosis (4.4%), 4 monoclonal gammopathy of undetermined significance (MGUS, 2.9%), and others. Serum-based MALDI-TOF-MS demonstrated a high detection rate for M-protein identification compared to SPEP, serum IFE, sFLC and urine IFE (98.5% vs. 75.9% vs. 86.9% vs. 71.5% vs. 76.0%). Plasma-based testing achieved a concordance rate of 89.1% (122/137). Using IFE/sFLC results as the gold standard, the sensitivity of MALDI-TOF-MS for the identification of κ and λ light chains (LC) was 75.8% and 80.0%, respectively. For IgG and IgA identification, the sensitivity of MALDI-TOF-MS was 93.5% (58/62) and 65.5% (19/29), respectively. Additionally, MALDI-TOF-MS detected LC glycosylation in 17 patients and other post-translational modifications (PTMs) in 4 patients. Notably, post-treatment monitoring revealed that two patients with eliminated glycosylation peaks achieved complete response, while one with persistent glycosylation had a very good partial response. CONCLUSIONS: MALDI-TOF-MS is a reliable tool for M-protein detection, offering high detection rate, LC glycosylation identification, and PTM analysis. Additionally, in a small cohort, we observed that changes in the abnormal peaks detected by MALDI-TOF-MS may correlate with treatment response.
Common Questions
What is Amyloidosis?
Amyloidosis is a group of conditions where abnormal proteins called amyloid fibres build up in organs, causing progressive organ damage. ATTR amyloidosis (transthyretin) and AL amyloidosis are the most common types. New RNA-based therapies and small molecule stabilisers have significantly improved outcomes.
How many clinical trials are available for Amyloidosis?
RareWays currently indexes 466 clinical trials for Amyloidosis, of which 158 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Amyloidosis 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.