Drug repurposing using real-world data
Highlights•250 records, including 144 original studies, met the eligibility criteria for inclusion
in this review.•Studies evaluated repurposing mainly for cardiovascular disease, COVID-19 …
in this review.•Studies evaluated repurposing mainly for cardiovascular disease, COVID-19 …
Children's interstitial and diffuse lung disease
S Cunningham, A Jaffe, LR Young - The Lancet Child & Adolescent …, 2019 - thelancet.com
Children's interstitial and diffuse lung disease (chILD) is a rare heterogenous group of
conditions, with symptoms often overlapping with more common conditions, impeding …
conditions, with symptoms often overlapping with more common conditions, impeding …
Improvements needed to support people living and working with a rare disease in Northern Ireland: current rare disease support perceived as inadequate
J McMullan, AL Crowe, C Bailie, K Moore… - Orphanet Journal of …, 2020 - Springer
Background Many people living and working with rare diseases describe consistent
difficulties accessing appropriate information and support. In this study an evaluation of the …
difficulties accessing appropriate information and support. In this study an evaluation of the …
[HTML][HTML] Protocol for the EARCO Registry: a pan-European observational study in patients with α1-antitrypsin deficiency
T Greulich, A Altraja, M Barrecheguren… - ERJ open …, 2020 - Eur Respiratory Soc
Rationale and objectives Alpha-1 antitrypsin deficiency (AATD) is a genetic condition that
leads to an increased risk of emphysema and liver disease. Despite extensive investigation …
leads to an increased risk of emphysema and liver disease. Despite extensive investigation …
Demographic and clinical characteristics of patients with α1-antitrypsin deficiency genotypes PI* ZZ and PI* SZ in the Spanish registry of EARCO
M Torres-Durán, JL López-Campos… - ERJ Open …, 2022 - Eur Respiratory Soc
Background The Spanish registry of α1-antitrypsin deficiency (AATD) integrated in the
European Alpha-1 Research Collaboration (EARCO) provides information about the …
European Alpha-1 Research Collaboration (EARCO) provides information about the …
The importance of reference centers and registries for rare diseases: the example of alpha-1 antitrypsin deficiency
M Miravitlles, A Nunez, M Torres-Duran… - COPD: Journal of …, 2020 - Taylor & Francis
Abstract Alpha-1 antitrypsin deficiency (AATD) is a rare and underdiagnosed disease that is
associated with the development of liver disease in adults and children and pulmonary …
associated with the development of liver disease in adults and children and pulmonary …
[HTML][HTML] A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem
TL Klein, J Bender, S Bolton… - Orphanet Journal of …, 2024 - ncbi.nlm.nih.gov
People with rare lysosomal storage diseases face challenges in their care that arise from
disease complexity and heterogeneity, compounded by many healthcare professionals …
disease complexity and heterogeneity, compounded by many healthcare professionals …
[HTML][HTML] Long-term safety study of colistimethate sodium (Colobreathe®): Findings from the UK Cystic Fibrosis Registry
S Kaplan, A Lee, N Caine, SC Charman… - Journal of Cystic …, 2021 - Elsevier
Background As part of the risk management plan in Europe, a long-term observational study
was conducted to monitor the safety of colistimethate sodium dry powder for inhalation (CMS …
was conducted to monitor the safety of colistimethate sodium dry powder for inhalation (CMS …
Swapping data: A pragmatic approach for enabling academic-industrial partnerships
J Kasprzak, S Frey, H Oetlinger… - Digital …, 2023 - journals.sagepub.com
Objectives Academic institutions have access to comprehensive sets of real-world data.
However, their potential for secondary use—for example, in medical outcomes research or …
However, their potential for secondary use—for example, in medical outcomes research or …
How to establish and sustain a disease register: Insights from a qualitative study of six registers in the UK
E Stubbs, J Exley, R Wittenberg, N Mays - 2024 - researchsquare.com
Background: The advent of new chronic conditions such as long COVID-19 raises the
question of whether and, if so, how best to establish new disease registers for such …
question of whether and, if so, how best to establish new disease registers for such …