Editorial: Spinal Muscular Atrophy: Evolutions and Revolutions of Modern Therapy

por Finkel, Richard S., Schara-Schmidt, Ulrike, Hagenacker, Tim
Publicado 2020

Evolution of Next Generation Therapeutics: Past, Present, and Future of Precision Medicines

por Ramamoorthy, Anuradha, Karnes, Jason H., Finkel, Richard, Blanchard, Rebecca, Pacanowski, Michael
Publicado 2019

The Use of Autologous Blood Patch in Ullrich Muscular Dystrophy and Recurrent Pneumothorax

por Navas Nazario, Aledie, Cooper, Felicia I, Weber-Guzman, Fabiola, Finkel, Richard S
Publicado 2022

SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

por Kayadjanian, Nathalie, Burghes, Arthur, Finkel, Richard S, Mercuri, Eugenio, Rouault, Francoise, Schwersenz, Inge, Talbot, Kevin
Publicado 2013

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials

por Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah
Publicado 2019

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

por Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.
Publicado 2021

Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis

por Coratti, Giorgia, Cutrona, Costanza, Pera, Maria Carmela, Bovis, Francesca, Ponzano, Marta, Chieppa, Fabrizia, Antonaci, Laura, Sansone, Valeria, Finkel, Richard, Pane, Marika, Mercuri, Eugenio
Publicado 2021

Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model

por MacCannell, Drew, Berger, Zdenek, Kirschner, Janbernd, Mercuri, Eugenio, Farrar, Michelle A., Iannaccone, Susan T., Kuntz, Nancy L., Finkel, Richard S., Valente, Marta, Muntoni, Francesco
Publicado 2022

Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United States

por Darras, Basil T., Guye, Sabrina, Hoffart, Janine, Schneider, Sophie, Gravestock, Isaac, Gorni, Ksenija, Fuerst‐Recktenwald, Sabine, Scalco, Renata S., Finkel, Richard S., De Vivo, Darryl C.
Publicado 2022

Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients

por Lochmüller, Hanns, Evans, David, Farwell, Wildon, Finkel, Richard, Goemans, Nathalie, de Lemus, Mencia, Matyushenko, Vitaliy, Muntoni, Francesco, Ouillade, Marie-Christine, Schwersenz, Inge, Wilson, Petra
Publicado 2018

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

por Finkel, Richard S., Flanigan, Kevin M., Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H. Lee, Reha, Allen, Northcutt, Valerie J., Elfring, Gary, Barth, Jay, Peltz, Stuart W.
Publicado 2013

Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

por Finanger, Erika, Vandenborne, Krista, Finkel, Richard S., Lee Sweeney, H., Tennekoon, Gihan, Yum, Sabrina, Mancini, Maria, Bista, Pradeep, Nichols, Andrew, Liu, Hanlan, Fretzen, Angelika, Donovan, Joanne M.
Publicado 2019

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy

por Day, John W., Mendell, Jerry R., Mercuri, Eugenio, Finkel, Richard S., Strauss, Kevin A., Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P., Chand, Deepa H.
Publicado 2021

Longitudinal natural history of type I spinal muscular atrophy: a critical review

por Mercuri, Eugenio, Lucibello, Simona, Perulli, Marco, Coratti, Giorgia, de Sanctis, Roberto, Pera, Maria Carmela, Pane, Marika, Montes, Jacqueline, de Vivo, Darryl C., Darras, Basil T., Kolb, Stephen J., Finkel, Richard S.
Publicado 2020

Scientific rationale for a higher dose of nusinersen

por Finkel, Richard S., Ryan, Monique M., Pascual Pascual, Samuel Ignacio, Day, John W., Mercuri, Eugenio, De Vivo, Darryl C., Foster, Richard, Montes, Jacqueline, Gurgel‐Giannetti, Juliana, MacCannell, Drew, Berger, Zdenek
Publicado 2022

Nusinersen Treatment of Children with Later-Onset Spinal Muscular Atrophy and Scoliosis Is Associated with Improvements or Stabilization of Motor Function †

por Dunaway Young, Sally, Montes, Jacqueline, Glanzman, Allan M., Gee, Richard, Day, John W., Finkel, Richard S., Darras, Basil T., De Vivo, Darryl C., Gambino, Giulia, Foster, Richard, Wong, Janice, Garafalo, Steve, Berger, Zdenek
Publicado 2023

DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results

por Finkel, Richard S., Day, John W., Pascual Pascual, Samuel Ignacio, Ryan, Monique M., Mercuri, Eugenio, De Vivo, Darryl C., Montes, Jacqueline, Gurgel-Giannetti, Juliana, Monine, Michael, Gambino, Giulia, Makepeace, Corinne, Foster, Richard, Berger, Zdenek
Publicado 2023