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- [41] Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)Journal of Neurology, 2023, 270 : 2547 - 2549Maryam Oskoui论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryJohn W. Day论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryNicolas Deconinck论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryElena S. Mazzone论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryAndres Nascimento论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryKayoko Saito论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryCarole Vuillerot论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryGiovanni Baranello论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryNathalie Goemans论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryJanbernd Kirschner论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryAnna Kostera-Pruszczyk论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryLaurent Servais论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryGergely Papp论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryKsenija Gorni论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryHeidemarie Kletzl论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryCarmen Martin论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryTammy McIver论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryRenata S. Scalco论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryHannah Staunton论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryWai Yin Yeung论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryPaulo Fontoura论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and NeurosurgeryEugenio Mercuri论文数: 0 引用数: 0 h-index: 0机构: McGill University,Departments of Pediatrics and Neurology and Neurosurgery
- [42] Non-diabetic Euglycemic Ketoacidosis in an Adult Patient With Spinal Muscular Atrophy Type IICUREUS JOURNAL OF MEDICAL SCIENCE, 2023, 15 (09)Khludenev, George论文数: 0 引用数: 0 h-index: 0机构: Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USA Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USALe Cam, Elise论文数: 0 引用数: 0 h-index: 0机构: Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USA Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USAAinapurapu, Bujji B.论文数: 0 引用数: 0 h-index: 0机构: Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USA Univ Arizona, Coll Med Tucson, Internal Med, Tucson, AZ 85721 USA
- [43] Clinical Course in a Patient With Spinal Muscular Atrophy Type 0 Treated With Nusinersen and Onasemnogene AbeparvovecJOURNAL OF CHILD NEUROLOGY, 2020, 35 (11) : 717 - 723Matesanz, Susan E.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USACurry, Candace论文数: 0 引用数: 0 h-index: 0机构: Mission Childrens Specialists, Neurol, Asheville, NC USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USAGross, Brianna论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USARubin, Adam I.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Pathol & Lab Med, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Dermatol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USALinn, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pathol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USAYum, Sabrina W.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA Univ Penn, Div Neurol, Perelman Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USAKichula, Elizabeth A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA Univ Penn, Div Neurol, Perelman Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Neurol, 3401 Civ Ctr Blvd, Philadelphia, PA 19104 USA
- [44] RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)NEUROMUSCULAR DISORDERS, 2023, 33 : S88 - S88Finkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAFarrar, M.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp, Sydney, NSW, Australia UNSW Sydney, UNSW Med, Sydney, NSW, Australia St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Paediat, MDUK Oxford Neuromuscular Ctr, Oxford, England Univ Hosp Liege, Div Child Neurol, Dept Paediat, Ctr References Malad Neuromusculaires, Liege, Belgium Univ Liege, Liege, Belgium Hop Armand Trousseau, AP HP, I Mot Inst Myol, Paris, France St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAVlodavets, D.论文数: 0 引用数: 0 h-index: 0机构: Russian Nat Res Med Univ, Veltischev Clin Paediat Res Inst Pirogov, Russian Children Neuromusc Ctr, Moscow, Russia St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAZanoteli, E.论文数: 0 引用数: 0 h-index: 0机构: Univ Sao Paulo, Dept Neurol, Fac Med, Sao Paulo, SP, Brazil St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAAl-Muhaizea, M.论文数: 0 引用数: 0 h-index: 0机构: King Faisal Specialist Hosp & Res Ctr, Dept Neurosci, Riyadh, Saudi Arabia Res Ctr Riyadh, Riyadh, Saudi Arabia St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAPrufer, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio de Janeiro, Pediat Dept, Fac Med, Rio De Janeiro, Brazil St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USANelson, L.论文数: 0 引用数: 0 h-index: 0机构: UT SouthWestern Med Ctr, Phys Therapy, Dallas, TX USA St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAFischer, C.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAGerber, M.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev, Safety, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAGorni, K.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAKletzl, H.论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Develt, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAPalfreeman, L.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAGaki, E.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USAFontoura, P.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USABertini, E.论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Childrens Res Hosp IRCCS, Res Unit Neuromusc & Neurodegen Disorders, Rome, Italy St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA
- [45] Fertility outcomes in risdiplam-treated male patients with spinal muscular atrophy (SMA): a multicenter case seriesNEUROMUSCULAR DISORDERS, 2024, 43Erdler, M.论文数: 0 引用数: 0 h-index: 0机构: Clin Donaustadt, Dept Neurol, Vienna, Austria Clin Donaustadt, Dept Neurol, Vienna, AustriaCoskery, S.论文数: 0 引用数: 0 h-index: 0机构: Childrens Alabama, Birmingham, AL USA Clin Donaustadt, Dept Neurol, Vienna, AustriaFrey, M.论文数: 0 引用数: 0 h-index: 0机构: Mem Healthcare Inst Neurosci, Owosso, MI USA Clin Donaustadt, Dept Neurol, Vienna, AustriaLopez, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Heersink Sch Med, Dept Pediat, Div Pediat Neurol, Birmingham, AL USA Clin Donaustadt, Dept Neurol, Vienna, Austria
- [46] Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)EXPERT OPINION ON ORPHAN DRUGS, 2022, 10 (01): : 65 - 72Deconinck, N.论文数: 0 引用数: 0 h-index: 0机构: UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium Univ Libre Bruxelles ULB, Queen Fabiola Childrens Univ Hosp, Neuromuscular Reference Ctr, Brussels, Belgium Univ Libre Bruxelles ULB, Queen Fabiola Childrens Univ Hosp, Paediat Neurol Dept, Brussels, Belgium Hop Univ Enfants Reine Fabiola, Dept Pediat Neurol, Ave Jean Joseph Crocq 15, B-1020 Brussels, Belgium UZ Gent, Neuromuscular Reference Ctr, Ghent, BelgiumDevos, E.论文数: 0 引用数: 0 h-index: 0机构: UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium UZ Gent, Neuromuscular Reference Ctr, Ghent, Belgium
- [47] Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal StudyMUSCLE & NERVE, 2025, 71 (03) : 384 - 391Gavriilaki, Maria论文数: 0 引用数: 0 h-index: 0机构: Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, Greece Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, GreeceMoschou, Maria论文数: 0 引用数: 0 h-index: 0机构: Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, Greece Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, GreecePagiantza, Maria论文数: 0 引用数: 0 h-index: 0机构: Aristotle Univ Thessaloniki, Sch Med, Thessaloniki, Greece Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, GreeceArnaoutoglou, Marianthi论文数: 0 引用数: 0 h-index: 0机构: Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, Greece Aristotle Univ Thessaloniki, Ahepa Univ Hosp, Sch Med, Lab Clin Neurophysiol, Thessaloniki, Greece Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, GreeceKimiskidis, Vasilios论文数: 0 引用数: 0 h-index: 0机构: Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, Greece Aristotle Univ Thessaloniki, AHEPA Univ Hosp, Sch Med, Dept Neurol 1, Thessaloniki, Greece
- [48] Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH StudyNEUROLOGY AND THERAPY, 2023, 12 (02) : 543 - 557Chiriboga, Claudia A. A.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USA Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USABruno, Claudio论文数: 0 引用数: 0 h-index: 0机构: IRCCS Ist Giannina Gaslini, Ctr Translat & Expt Myol, Genoa, Italy Univ Genoa, Dept Neurosci Rehabil Ophthalmol Genet Maternal &, Genoa, Italy Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USADuong, Tina论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Neurol, Palo Alto, CA USA Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAFischer, Dirk论文数: 0 引用数: 0 h-index: 0机构: Univ Basel, Univ Childrens Hosp Basel, Div Neuropediat, Basel, Switzerland Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAMercuri, Eugenio论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Pediat Neurol Inst, Rome, Italy Fdn PoliclinicoGemelli IRCCS, Nemo Pediatr, Rome, Italy Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAKirschner, Janbernd论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Fac Med, Med Ctr, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAKostera-Pruszczyk, Anna论文数: 0 引用数: 0 h-index: 0机构: Med Univ Warsaw, Dept Neurol, Warsaw, Poland F Hoffmann La Roche Ltd, Prod Dev Neurosci, Basel, Switzerland Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAJaber, Birgit论文数: 0 引用数: 0 h-index: 0机构: Univ Coll London, NIHR Great Ormond St Hosp Biomed Res Ctr, Safety, Great Ormond St Inst Child Hlth, Basel, Switzerland Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAGorni, Ksenija论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAKletzl, Heidemarie论文数: 0 引用数: 0 h-index: 0机构: Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USACarruthers, Imogen论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, England Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAMartin, Carmen论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, England Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAWarren, Francis论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, England Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAScalco, Renata S. S.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAWagner, Kathryn R. R.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USAMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, 180 Ft Washington Ave 552, New York, NY 10032 USA
- [49] Safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)NEUROMUSCULAR DISORDERS, 2023, 33 : S92 - S93Baranello, G.论文数: 0 引用数: 0 h-index: 0机构: UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, England Gt Ormond Str Hosp Trust, London, England Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandChiriboga, C.论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Dept Neurol, Irving Med Ctr, New York, NY USA UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandServais, L.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Paediat, MDUK Oxford Neuromusc Ctr, Oxford, England Univ Hosp Liege, Div Child Neurol, Dept Paediat, Ctr References Malad Neuromusculaires, Liege, Belgium Univ Liege, Liege, Belgium Hop Armand Trousseau, AP HP, I Mot, Inst Myol, Paris, France UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandDarras, B.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA USA UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandDay, J.论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Neurol, Palo Alto, CA USA UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandDeconinck, N.论文数: 0 引用数: 0 h-index: 0机构: Univ Libre Bruxelles, Queen Fabiola Childrens Univ Hosp, Ctr Reference Malad Neuromusculaires, Brussels, Belgium UZ Gent, Neuromusc Reference Ctr, Ghent, Belgium UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandFarrar, M.论文数: 0 引用数: 0 h-index: 0机构: Sydney Childrens Hosp Network, Sydney, NSW, Australia UNSW Sydney, UNSW Med, Sydney, NSW, Australia UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandFinkel, R.论文数: 0 引用数: 0 h-index: 0机构: St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, Memphis, TN USA UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandBertini, E.论文数: 0 引用数: 0 h-index: 0机构: Bambino Gesu Childrens Res Hosp IRCCS, Res Unit Neuromusc & Neurodegen Disorders, Rome, Italy UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandKirschner, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Freiburg, Fac Med, Med Ctr, Dept Neuropaediat & Muscle Disorders, Freiburg, Germany UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandRasson, M.论文数: 0 引用数: 0 h-index: 0机构: Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandMazurkiewicz-Beldzinska, M.论文数: 0 引用数: 0 h-index: 0机构: Med Univ Gdansk, Dept Develt Neurol, Gdansk, Poland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandVlodavets, D.论文数: 0 引用数: 0 h-index: 0机构: Pirogov Russian Nat Res Med Univ, Russian Children Neuromusc Ctr, Veltischev Clin Paed Res Inst, Moscow, Russia UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandBader-Weder, S.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Safety, Basel, Switzerland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandGorni, K.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandJaber, B.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Safety, Basel, Switzerland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandYeung, W.论文数: 0 引用数: 0 h-index: 0机构: Roche Prod Ltd, Welwyn Garden City, Herts, England UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandPapp, G.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Safety, Basel, Switzerland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandScalco, R.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Neurol, Basel, Switzerland UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, EnglandMercuri, E.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Paediat Neurol Inst, Rome, Italy Fdn Policlin Gemelli IRCCS, Nemo Pediat, Rome, Italy UCL, NIHR Gt Ormond St Hosp, Dubowitz Neuromusc Ctr, Biomed Res Ctr,Gt Ormond St Inst Child Hlth, London, England
- [50] Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophyANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2021, 8 (01): : 54 - 65Sergott, Robert C.论文数: 0 引用数: 0 h-index: 0机构: Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USA Thomas Jefferson Univ, Annesley EyeBrain Ctr, Philadelphia, PA 19107 USA Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAAmorelli, Giulia M.论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Paediat Neurol & Nemo Ctr, Rome, Italy Policlin Gemelli, Rome, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USABaranello, Giovanni论文数: 0 引用数: 0 h-index: 0机构: UCL, Dubowitz Neuromuscular Ctr, NIHR Great Ormond St Hosp, Biomed Res Ctr,Great Ormond St Inst Child Hlth, London, England Great Ormond St Hosp Trust, London, England Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USABarreau, Emmanuel论文数: 0 引用数: 0 h-index: 0机构: Inst Myol, Paris, France Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USABeres, Shannon论文数: 0 引用数: 0 h-index: 0机构: Stanford Univ, Dept Ophthalmol, Dept Neurol, Palo Alto, CA 94304 USA Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAKane, Steven论文数: 0 引用数: 0 h-index: 0机构: Columbia Univ, Med Ctr, New York, NY USA Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAMercuri, Eugenio论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Paediat Neurol & Nemo Ctr, Rome, Italy Policlin Gemelli, Rome, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAOrazi, Lorenzo论文数: 0 引用数: 0 h-index: 0机构: Catholic Univ, Paediat Neurol & Nemo Ctr, Rome, Italy Policlin Gemelli, Rome, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USASantaMaria, Melissa论文数: 0 引用数: 0 h-index: 0机构: Thomas Jefferson Univ, Annesley EyeBrain Ctr, Philadelphia, PA 19107 USA Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USATremolada, Gemma论文数: 0 引用数: 0 h-index: 0机构: IRCCS Ist Auxol Italiano, Ophthalmol Dept, Neuroophthalmol Ctr, Milan, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USASantarsiero, Diletta论文数: 0 引用数: 0 h-index: 0机构: IRCCS Ist Auxol Italiano, Ophthalmol Dept, Neuroophthalmol Ctr, Milan, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAWaskowska, Agnieszka论文数: 0 引用数: 0 h-index: 0机构: Med Univ Gdansk, Dept Dev Neurol, Gdansk, Poland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAYashiro, Shigeko论文数: 0 引用数: 0 h-index: 0机构: Natl Ctr Global Hlth & Med NCGM, Dept Ophthalmol, Tokyo, Japan Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USADenk, Nora论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Pharmaceut Sci, Roche Innovat Ctr Basel, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAFurst-Recktenwald, Sabine论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Neurol, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAGerber, Marianne论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev, Safety, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAGorni, Ksenija论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, PDMA Neurosci & Rare Dis, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAJaber, Birgit论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev, Safety, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAJacobsen, Bjoern论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Pharmaceut Sci, Roche Innovat Ctr Basel, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAMueller, Lutz论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Pharmaceut Sci, Roche Innovat Ctr Basel, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USANave, Stephane论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Roche Pharma Res & Early Dev, Pharmaceut Sci, Roche Innovat Ctr Basel, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAScalco, Renata S.论文数: 0 引用数: 0 h-index: 0机构: F Hoffmann La Roche Ltd, Pharma Dev Neurol, Basel, Switzerland Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USAMarzoli, Stefania B.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Ist Auxol Italiano, Ophthalmol Dept, Neuroophthalmol Ctr, Milan, Italy Wills Eye Hosp & Res Inst, Dept Neuroophthalmol, Philadelphia, PA USA