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 …
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 …
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
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 …
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 …
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 …
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 …
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
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 …
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
Objective To evaluate the psychometric properties of the AL‐PROfile, a patient‐reported
outcome measure combining the Patient‐Reported Outcomes Measurement Information …
outcome measure combining the Patient‐Reported Outcomes Measurement Information …
[HTML][HTML] Renal Response Criteria for Clinical Trials in Amyloid Light Chain Amyloidosis
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 …
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 …
still have a multitude of different aspects in the past few decades. The range goes from …