Peripheral nervous, hepatic, and gastrointestinal endpoints for AL amyloidosis clinical trials: report from the amyloidosis forum multi-organ system working group

ML Mauermann, JO Clarke, WJ Litchy, L Obici… - Advances in …, 2023 - Springer
Systemic immunoglobulin light chain (AL) amyloidosis is a heterogeneous rare disease
driven by a destructive monoclonal gammopathy and typified by misfolded immunoglobulin …

The clinical trials landscape in immunoglobulin light chain amyloidosis: a systematic review

R Ramachandran, D Shah, C Luo, V Shah… - Blood …, 2024 - ashpublications.org
Immunoglobulin light chain (AL) amyloidosis is a rare hematologic disorder characterized by
end-organ damage from deposition of fibrillar aggregates of kinetically unstable light chains …

Functional status and quality of life in light-chain amyloidosis: advanced imaging, longitudinal changes, and outcomes

OF Clerc, S Vijayakumar, SAM Cuddy, G Bianchi… - Heart Failure, 2024 - jacc.org
Background In light-chain (AL) amyloidosis, whether functional status and heart failure–
related quality of life (HF-QOL) correlate with cardiomyopathy severity, improve with therapy …

Pathway for development and validation of multi-domain endpoints for amyloid light chain (AL) amyloidosis

J Signorovitch, J Zhang, D Brown, P Dunnmon… - … Innovation & Regulatory …, 2024 - Springer
Immunoglobin light chain (AL) amyloidosis is a rare disease in which a plasma cell
dyscrasia leads to deposition of insoluble amyloid fibrils in multiple organs. To facilitate …

Autologous stem cell transplantation in AL amyloidosis: Muddy waters

P Hagen, A D'Souza - Blood Reviews, 2024 - Elsevier
Immunoglobulin light chain (AL) amyloidosis is a malignant plasma cell dyscrasia causing
multi-organ morbidity. High dose melphalan and autologous stem cell transplantation …

Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings

MS Maurer, P Soman, A Hernandez, P Garcia-Pavia… - Advances in …, 2024 - Springer
Conclusion Given rapid evolution of natural history in ATTR amyloidosis, the utility of
historical control data is limited. Leveraging contemporary real-world data is essential for …

Differences in patient‐reported outcomes (PROs) by disease severity in light chain (AL) amyloidosis

A D'Souza, A Szabo, I Akinola… - European Journal of …, 2023 - Wiley Online Library
Objective To assess the impact of organ involvement on patient‐reported outcomes (PROs)
in light chain (AL) amyloidosis. Methods PROs were evaluated using the KCCQ‐12 …

Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

A D'Souza, A Szabo, I Akinola… - European Journal of …, 2024 - Wiley Online Library
Objective To evaluate the psychometric properties of the AL‐PROfile, a patient‐reported
outcome measure combining the Patient‐Reported Outcomes Measurement Information …

[HTML][HTML] Renal Response Criteria for Clinical Trials in Amyloid Light Chain Amyloidosis

N Leung, R Comenzo, J Gillmore, A Havasi… - Kidney International …, 2024 - Elsevier
Immunoglobin light chain (AL) amyloidosis is a rare disease characterized by organ
deposition of amyloid fibrils, most commonly in the heart and kidney. Disease heterogeneity …

Novel and Proven Models of Public, Private, and Public–Private Partnerships in Healthcare: An Update

HA Wieland, J Maas - 2024 - Springer
Initiatives to share assets in the life science sector through dedicated partnerships had and
still have a multitude of different aspects in the past few decades. The range goes from …