Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading

Authors: Erik Landfeldt, Alberto Alemán, Sophia Abner, Rongrong Zhang, Christian Werner, Ioannis Tomazos, Hanns Lochmüller, Ros M. Quinlivan, Karim Wahbi

Source: Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-34 (2024)

Subject Terms: Heart, Cardiomyopathy, Neuromuscular disease, Treatment, Guidelines, GRADE, Medicine

File Description: electronic resource

Relation: https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/c09edffea8ba4e5b8f6030cb32aaaa45

Lifetime impact of achondroplasia study in Europe (LIAISE): findings from a multinational observational study

Authors: Mohamad Maghnie, Oliver Semler, Encarna Guillen-Navarro, Angelo Selicorni, Karen E. Heath, Gabriele Haeusler, Lars Hagenäs, Andrea Merker, Antonio Leiva-Gea, Vanesa López González, Adalbert Raimann, Mirko Rehberg, Fernando Santos-Simarro, Diana-Alexandra Ertl, Pernille Axél Gregersen, Roberta Onesimo, Erik Landfeldt, James Jarrett, Jennifer Quinn, Richard Rowell, Jeanne Pimenta, Shelda Cohen, Thomas Butt, Renée Shediac, Swati Mukherjee, Klaus Mohnike

Source: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-19 (2023)

Subject Terms: Achondroplasia, Skeletal dysplasia, Fibroblast growth factor receptor 3 (FGFR3), Natural history, Disease burden, Medicine

File Description: electronic resource

Relation: https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/396b4128425f4efaa0a0c79776e03d51

Areas of improvement in the medical care of SMA: evidence from a nationwide patient registry in Germany

Authors: Berenike Leibrock, Erik Landfeldt, Justine Hussong, Tabea Huelle, Hannah Mattheus, Simone Thiele, Maggie C. Walter, Michael Zemlin, Eva Moehler, Ullrich Dillman, Sophia Abner, Marina Flotats-Bastardas

Source: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-10 (2023)

Subject Terms: (8/10): spinal muscular atrophy, Nusinersen, Risdiplam, Medical care, Rehabilitation, Non-invasive ventilation, Medicine

File Description: electronic resource

Relation: https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/3e8f7a0268f54429bfe7482d823e1c97

Correction: Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis.

Authors: Nermina Ferizovic, Jessica Summers, Igor Beitia Ortiz de Zárate, Christian Werner, Joel Jiang, Erik Landfeldt, Katharina Buesch

Source: PLoS ONE, Vol 19, Iss 12, p e0315682 (2024)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/279b3f01d1a64a0dbcb53b34df4086b7

Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis.

Authors: Nermina Ferizovic, Jessica Summers, Igor Beitia Ortiz de Zárate, Christian Werner, Joel Jiang, Erik Landfeldt, Katharina Buesch

Source: PLoS ONE, Vol 17, Iss 3, p e0265879 (2022)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/40ec53f8b01e432d9568f6f2553a52f2

Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy

Authors: Erik Landfeldt, Hanns Lochmüller, Peter Lindgren

Source: Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-3 (2019)

Subject Terms: Medicine

File Description: electronic resource

Relation: http://link.springer.com/article/10.1186/s13023-018-0975-3; https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/979b2fc01c574cc9868298d6c5f4ab0b

[Untitled]

Authors: Erik Landfeldt, Michelle Eagle, Volker Straub, Hanns Lochmüller, Katharine Bushby, Peter Lindgren

Source: Global & Regional Health Technology Assessment, Vol 4, Iss 1 (2017)

Subject Terms: Medical technology, R855-855.5

File Description: electronic resource

Relation: https://journals.aboutscience.eu/index.php/grhta/article/view/383; https://doaj.org/toc/2284-2403; https://doaj.org/toc/2283-5733

Access URL: https://doaj.org/article/d0310916b26b42e3b807ac00323657ef

Mortality cost of Duchenne muscular dystrophy

Authors: Erik Landfeldt, Michelle Eagle, Volker Straub, Hanns Lochmüller, Katharine Bushby, Peter Lindgren

Source: Global & Regional Health Technology Assessment, Vol 4, Iss 1, Pp e100-e103 (2017)

Subject Terms: Medical technology, R855-855.5

File Description: electronic resource

Relation: http://www.grhta.com/Attach/1442463D-F44E-4BCE-ADAE-8AF863A3B180/CE681EFC-A1CF-4248-9D95-51F60211EE1E; https://doaj.org/toc/2284-2403; https://doaj.org/toc/2283-5733

Access URL: https://doaj.org/article/6294035e85794de6a4806b0875086729

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