Sickle cell disease: a global patient registry review

O Borecka, A Ofori, F Amini, S Llewellyn - Future Rare Diseases, 2022 - Taylor & Francis
Sickle cell disease (SCD) is one of the most common inherited diseases. This review
identified and assessed key characteristics of the increasing number of SCD registries …

Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study

FP Thomas, M Saporta, S Attarian… - Neurodegenerative …, 2021 - Taylor & Francis
Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and sensory
neuropathy affecting the peripheral nervous system. This study will explore the real-world …

[PDF][PDF] The patient-reported impact of Charcot-Marie-Tooth disease in the real world: the design and conduct of a digital lifestyle study (CMT&Me)

FP Thomas, M Saporta, S Attarian, T Sevilla… - 2020 - scholar.archive.org
Background Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and
sensory neuropathy that affects the peripheral nervous system, leading to progressive …