Results 81 to 90 of about 2,159 (173)

Zolgensma : A Curative Treatment for a Severely Debilitating Disease

Asian Journal of Pharmaceutical and Health Sciences, 2022
Annmaria Tom, Aruna T, Roshni M.C, Ashly Santhosh K, Krishna P, Kiron SS   +5 more
openaire   +1 more source

La terapia génica de la atrofia muscular espinal. Una revisión sistemática [PDF]

, 2022
La atrofia muscular espinal es una enfermedad hereditaria crónica y degenerativa con una alta incidencia en la población, con una morbimortalidad elevada en pacientes pediátricos y elevados costes de recursos sanitarios en pacientes que llegan a la ...
León Martínez, Juan Ignacio
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Custo-efetividade do Zolgensma® na América Latina: uma revisão da literatura

CONTRIBUCIONES A LAS CIENCIAS SOCIALES
O objetivo deste trabalho foi sumarizar estudos farmacoeconômicos de custo-efetividade do tratamento farmacológico da atrofia muscular espinhal (AME) tipo I, em pacientes até dois anos de idade com o medicamento Zolgensma®, em países da América Latina (AL). Realizou-se uma busca na literatura científica, entre o período de janeiro de 2019 a dezembro de
Virgínia de Castro Rodrigues, Gabriela Bittencourt González Mosegui, Cid Manso de Mello Vianna   +2 more
openaire   +1 more source

Motor and neurocognitive profiles of children with symptomatic spinal muscular atrophy type 1 with two copies of SMN2 before and after treatment: a longitudinal observational study

Frontiers in Neurology
IntroductionSpinal muscular atrophy (SMA) is a neurodegenerative disease caused by mutations in the survival motor neuron 1 (SMN1) gene. In clinical studies, gene replacement therapy with onasemnogene abeparvovec (formerly AVXS-101, Zolgensma®, Novartis)
Ilaria Bitetti, Maria Rosaria Manna, Roberto Stella, Antonio Varone   +3 more
doaj   +1 more source

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational studyResearch in context

The Lancet Regional Health. Europe
Summary: Background: Real-world data on gene addition therapy (GAT) with onasemnogene abeparvovec (OA), including all age groups and with or without symptoms of the disease before treatment are needed to provide families with evidence-based advice and ...
Claudia Weiß, Lena-Luise Becker, Johannes Friese, Astrid Blaschek, Andreas Hahn, Sabine Illsinger, Oliver Schwartz, Günther Bernert, Maja von der Hagen, Ralf A. Husain, Klaus Goldhahn, Janbernd Kirschner, Astrid Pechmann, Marina Flotats-Bastardas, Gudrun Schreiber, Ulrike Schara, Barbara Plecko, Regina Trollmann, Veronka Horber, Ekkehard Wilichowski, Matthias Baumann, Andrea Klein, Astrid Eisenkölbl, Cornelia Köhler, Georg M. Stettner, Sebahattin Cirak, Oswald Hasselmann, Angela M. Kaindl, Sven F. Garbade, Jessika Johannsen, Andreas Ziegler, Petra Baum, Manuela Baumgartner, Astrid Bertsche, Markus Blankenburg, Jonas Denecke, Marcus Deschauer, Matthias Eckenweiler, Tobias Geis, Klaus Goldhahn, Martin Groß, René Günther, Tim Hagenacker, Eckard Hamelmann, Ralf A. Husain, Christoph Kamm, Birgit Kauffmann, Jan Christoph Koch, Wolfgang Löscher, Albert Ludolph, Pascal Martin, Alexander Mensch, Gerd Meyer zu Hörste, Christoph Neuwirth, Susanne Petri, Manuel Pühringer, Imke Rathmann, Dorothee Schäfer, Mareike Schimmel, Bertold Schrank, Olivia Schreiber-Katz, Anette Schwerin-Nagel, Martin Smitka, Meike Steinbach, Elisabeth Steiner, Johannes Stoffels, Manuela Theophil, Raffi Topakian, Matthias Türk, Matthias Vorgerd, Maggie C. Walter, Markus Weiler, Gert Wiegand, Gilbert Wunderlich, Claudia Diana Wurster, Daniel Zeller, Moritz Metelmann, Fiona Zeiner, Veronika Pilshofer, Mika Rappold, Josefine Pauschek, Christof Reihle, Annette Karolin Homma, Paul Lingor, Bettina Henzi, Elisabeth Steiner, Tabea Reinhardt, Dorothea Holzwarth, Wolfgang Wittmann, Manuela Theophil, Stefan Kappel, Maren Freigang, Benjamin Stolte, Kyriakos Martakis, Georg Classen, Doris Roland-Schäfer, Daniela Steuernagel, Hans Hartmann, Sophie Fischer, Marieke Wermuth, Mohamad Tareq Muhandes, Anna Hotter, Zeljko Uzelac, Steffen Naegel, Sarah Wiethoff, Nathalie Braun, Bogdan Bjelica, Heike Kölbel, Daniela Angelova-Toshkina, Bernd Wilken, Alma Osmanovic, Barbara Fiedler, Barbara Plecko, Maike Tomforde, Thomas Voelkl, Arpad von Moers, Petra Müller, Bettina Behring, Anne Güttsches, Peter Reilich, Wolfgang Wick, Corinna Stoltenburg, Simon Witzel, Julia Bellut, Georg Friedrich Hoffmann, Wolfgang Löscher, Kathrin Mörtlbauer, Alexandra Ille, Michael Schroth, Joenna Driemeyer, Luisa Semmler, Cornelia Müller, Katharina Dörnbrack, Manuel Pühringer, Michael Zemlin, Stephanie Geitmann, Hanna Sophie Lapp, Svenja Brakemeier, Tascha Gehrke, Klearchos Ntemiris, Nadja Kaiser, Sabine Borowski, Barbara Ramadan, Ulf Hustedt, Tobias Baum, Simon Witzel, Ilka Schneider, Esra Akova-Oztürk, Katharina Vill, Zylfie Dibrani, Camilla Wohnrade, Adela Della-Marina, Lisa Jung, Timo Deba, Joachim Zobel, Jens Schallner, Christina Kraut, Klaus Goldhahn, Peter Vollmann, Stephanie Schüssler, Melanie Roeder, Miriam Hiebeler, Nicole Berberich, Joanna Schneider, Zeljko Uzelac, Brigitte Brauner, Stefan Kölker, Elke Pernegger, Magdalena Gosk-Tomek, Sarah Braun, Deike Weiss, Gerrit Machetanz, Thorsten Langer, Christina Saier, Sandra Baumann, Sabine Hettrich, Gabriel Dworschak, Katharina Müller-Kaempffer, Isabelle Dittes, Andreas Thimm, Lisa Quinten, Kristina Albers, Andrea Bevot, Christa Bretschneider, Johannes Dorst, Thomas Kendzierski, Iris Hannibal, Jasmin Bischofberger, Tilman Riesmeier, Andrea Gangfuß, Eva Johann to Settel, Michael Grässl, Susan Fiebig, Carmen Hollerauer, Lea Seeber, Ina Krahwinkler, Irene Lange, Federica Montagnese, Marcel Mann-Richter, Alexandra Wagner, Johannes Dorst, Christine Leypold, Afshin Saffari, Elmecker Anna, Anna Wiesenhofer, Eva-Maria Wendel, Paula-Sophie Steffens, Sabine Wider, Adrian Tassoni, Andrea Dall, Franziska Busch, Daniela Zeisler, Maria Wessel, Jaqueline Lipka, Andrea Hackemer, Loreen Plugge, Eva Jansen, Erdmute Roth, Joachim Schuster, Anna Koelsch, Birgit Warken-Madelung, Michaela Schwippert, Britta Holtkamp, Katja Köbbing, Sander Claeys, Sandy Foerster, Daniela Zeisler, Simone Thiele, Heidi Rochau-Trumpp, Annette George, Joachim Schuster, Moritz Niesert, Tanja Neimair, Katia Vettori, Julia Haverkamp, Jila Taherpour, Juliane Hug, Franziska Wenzel, Christina Bant, Ute Baur, Kathrin Bühner, Melina Schlag, Lena Ruß, Hanna Küpper, Anja Müller, Kurt Wollinsky, Therese Well, Antonia Leinert, Barbara Andres, Heymut Omran, Nicole Claus, Kathrin Bühner, Anna Hagenmeyer, Marion Schnurr, Vladimir Dukic, Albert Christian Ludolph, Sabine Specht, Verena Angermair, Anna Hüpper, Daniela Banholzer, Sabine Stein, Tim Kampowski, Marion Richmann, Sylke Nicolai, Omar Atta, Birgit Meßmer, Heike de Vries, Elisabeth Rotenfusser, Alma Oscmanovic, Isabelle Renger, Hélène Guillemot, Ilka Lehnert, Mike Grünwedel, Laura Grimm, Guido Stocker, Angela M. Kaindl, Kurt Wollinsky, Annegret Hoevel, Theresa Stadler, Michal Fischer, Sibylle Vogt, Axel Gebert, Maggie C. Walter, Susanne Goldbach, Tim Hagenacker, Janbernd Kirschner, Hanns Lochmüller, Wolfgang Müller-Felber, Astrid Pechmann, Ulrike Schara-Schmidt, Kristina Probst-Schendzielorz, Annina Lang, Maren Nitzsche, Olivia Schreiber-Katz, Julie Hammer, Katharina Müller-Kaempfer, Corinna Wirner-Piotrowski, Lieske van der Stam, Anke Bongartz, Susanne Goldbach, Hanns Lochmüller, Cornelia Enzmann, Joël Fluss, Elea Galiart, Bettina Henzi, David Jacquier, Dominique Baumann Metzler, Anne Tscherter, Kristina Probst-Schendzielorz   +308 more
doaj   +1 more source

PE-094 O acordo de acesso gerenciado do onasemnogeno abeparvoveque trará economia para o Sistema Único de Saúde (SUS)? Uma análise de impacto orçamentário

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução (com objetivo): Em março de 2025, foi formalizado um Acordo de Acesso Gerenciado (AAG)  entre o Ministério da Saúde e a empresa detentora do registro do onasemnogeno abeparvoveque (OA) para o tratamento de pacientes com Atrofia Muscular ...
Carolina Felix Fortis, Bruna Bento dos Santos, Camila de Oliveira Cezar Faria, Ida Vanessa Doederlein Schwartz, Helena Nunes Cirilo   +4 more
doaj   +1 more source

Variation in Health Technology Assessment recommendations and reimbursement of Advanced Therapy Medicinal Products across nine european countries

Discover Health Systems
Background Advanced Treatment Medicinal Products (ATMPs) are innovative treatments aimed at severe diseases. However, patient access varies across European countries. The present research reviews the reimbursement status of the licensed ATMPs in England,
Paraskevi M. Georgiou, Kostas P. Athanasakis   +1 more
doaj   +1 more source

Kostprijs-plusmodel geeft inzicht in reële prijzen gentherapieën

, 2022
Goed nieuws voor patiënten met de zeldzame erfelijke spierziekte spinale musculaire atrofie (SMA) en de zeldzame erfelijke stofwisselingsziekte metachromatische leukodystrofie (MLD).
Ten Ham, Renske M.T., Heine, Renaud J.S.D., Van Den Berg, Sibren, Uyl-De Groot, Carin A., Thielen, Frederick W.   +4 more
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Onasemnogene abeparvovec (Zolgensma®) for the treatment of spinal muscular atrophy: A Protocol for Systematic Review

This project contains the protocol, search strategies, study selection materials, data extraction forms, risk-of-bias assessments, GRADE evidence profiles, PRISMA flow diagram, and manuscript files for a rapid systematic review and health technology ...
LINA SOFIA MORON DUARTE, Kelly Chacon, Ana Maria Rojas, Maria Paula Gutierrez, Adriana Rodriguez   +4 more
core   +1 more source

Zugangsgerechtigkeit von Therapien für seltene Erkrankungen: Eine ethische Analyse am Beispiel der spinalen Muskelatrophie [PDF]

Diese Bachelorarbeit untersucht die Zugangsgerechtigkeit von Therapien für seltene Erkrankungen, fokussiert auf die spinale Muskelatrophie (SMA) und das Medikament Zolgensma.
Peters, Helen
core   +1 more source