Spinal müsküler atrofi (Turkish Wikipedia)

Analysis of information sources in references of the Wikipedia article "Spinal müsküler atrofi" in Turkish language version.

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98nih.gov

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44web.archive.org

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12archive.org

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5worldcat.org

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3smanewstoday.com

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3curesma.org

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3europa.eu

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3fda.gov

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2rarediseases.org

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1nhs.uk

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1medlineplus.gov

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1childrenshospital.org

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1childrensnational.org

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1elsevier.com

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1sma-screening-alliance.org

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1medicines.org.uk

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1gene.com

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1uptodate.com

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1archives-ouvertes.fr

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1fiercebiotech.com

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1afm-telethon.fr

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1unimi.it

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1cytokinetics.com

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1businesswire.com

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1treat-nmd.eu

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acog.org

"Carrier Screening in the Age of Genomic Medicine – ACOG". www.acog.org. 25 Şubat 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 24 Şubat 2017.

afm-telethon.fr

amyotrophies-spinales.blogs.afm-telethon.fr

"Riluzole: premiers résultats décevants" (Fransızca). AFM Téléthon. 22 Eylül 2010. 8 Aralık 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 16 Mart 2017.

afp.com

"Spinraza (Nusinersen) Approved in the European Union as First Treatment for Spinal Muscular Atrophy". Agence France-Presse (AFP). 1 Haziran 2017. 4 Haziran 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 1 Haziran 2017.

archive.org

"Spinal muscular atrophy type 1: A noninvasive respiratory management approach". Chest. 117 (4): 1100-5. April 2000. doi:10.1378/chest.117.4.1100. PMID 10767247.
"Long-term survival in Werdnig-Hoffmann disease". American Journal of Physical Medicine & Rehabilitation. 86 (5): 339-45 quiz 346-8, 379. May 2007. doi:10.1097/PHM.0b013e31804a8505. PMID 17449977.
"Prevalence and risk factors for feeding and swallowing difficulties in spinal muscular atrophy types II and III". The Journal of Pediatrics. 160 (3): 447-451.e1. March 2012. doi:10.1016/j.jpeds.2011.08.016. PMID 21924737.
"Fatty acid oxidation abnormalities in childhood-onset spinal muscular atrophy: primary or secondary defect(s)?". Pediatric Neurology. 12 (1): 21-30. January 1995. doi:10.1016/0887-8994(94)00100-G. PMID 7748356.
"Behavioural problems in children and adolescents with spinal muscular atrophy and their siblings". Developmental Medicine and Child Neurology. 45 (1): 44-9. January 2003. doi:10.1017/S0012162203000082. PMID 12549754.
"Medical considerations of long-term survival of Werdnig-Hoffmann disease". American Journal of Physical Medicine & Rehabilitation. 86 (5): 349-55. May 2007. doi:10.1097/PHM.0b013e31804b1d66. PMID 17449979.
"Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy". Neurology. 68 (1): 51-5. January 2007. doi:10.1212/01.wnl.0000249142.82285.d6. PMID 17082463.
"Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells". Annals of Neurology. 58 (2): 194-202. August 2005. doi:10.1002/ana.20548. PMID 16049920.
"Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy". Neurology. 75 (24): 2190-7. December 2010. doi:10.1212/WNL.0b013e3182020332. PMID 21172842.
"Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition". Annals of Neurology. 64 (4): 465-70. October 2008. doi:10.1002/ana.21449. PMID 18661558.
"Efficacy of thyrotropin-releasing hormone in the treatment of spinal muscular atrophy". Journal of Child Neurology. 9 (3): 287-9. July 1994. doi:10.1177/088307389400900313. PMID 7930408.
"Riluzole attenuates spinal muscular atrophy disease progression in a mouse model". Muscle & Nerve. 28 (4): 432-7. October 2003. doi:10.1002/mus.10455. PMID 14506714.

archives-ouvertes.fr

hal.archives-ouvertes.fr

"Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design" (PDF). Journal of Medical Genetics. 47 (12): 856-8. December 2010. doi:10.1136/jmg.2010.080366. PMID 20837492. 10 Mart 2022 tarihinde kaynağından arşivlendi (PDF). Erişim tarihi: 26 Nisan 2023.

businesswire.com

"Scholar Rock Announces Positive 12-Month Top-Line Results From the TOPAZ Phase 2 Clinical Trial Evaluating Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy (SMA)". www.businesswire.com (İngilizce). 6 Nisan 2021. 6 Nisan 2021 tarihinde kaynağından arşivlendi. Erişim tarihi: 13 Mayıs 2021.

childrenshospital.org

"Spinal Muscular Atrophy (SMA) | Boston Children's Hospital". www.childrenshospital.org. 7 Eylül 2015 tarihinde kaynağından arşivlendi. Erişim tarihi: 25 Ekim 2020.

childrensnational.org

"Spinal Muscular Atrophy – Conditions | Children's National". childrensnational.org. 22 Eylül 2020 tarihinde kaynağından arşivlendi. Erişim tarihi: 25 Ekim 2020.

curesma.org

"Newborn Screening for Spinal Muscular Atrophy". Cure SMA (İngilizce). 14 Ocak 2020 tarihinde kaynağından arşivlendi. Erişim tarihi: 4 Mayıs 2020. Yazar |ad1= eksik |soyadı1= (yardım)
"SMA CARE SERIES – Musculoskeletal System" (PDF). www.curesma.org. Yaz 2017. 19 Şubat 2018 tarihinde kaynağından (PDF) arşivlendi. Erişim tarihi: 7 Aralık 2017. Yazar |ad1= eksik |soyadı1= (yardım)
"Novartis Releases Update on LMI070 (Branaplam) Clinical Trial". CureSMA. 25 Kasım 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 7 Ekim 2017.

cytokinetics.com

"CK-2127107". 16 Ekim 2014 tarihinde kaynağından arşivlendi.

doi.org

Verhaart (July 2017). "A multi-source approach to determine SMA incidence and research ready population". Journal of Neurology (İngilizce). 264 (7): 1465-1473. doi:10.1007/s00415-017-8549-1. ISSN 0340-5354. PMC 5502065 $2. PMID 28634652.
"Ramblings in the history of spinal muscular atrophy". Neuromuscular Disorders (İngilizce). 19 (1): 69-73. 2009. doi:10.1016/j.nmd.2008.10.004. PMID 18951794. 28 Mart 2023 tarihinde kaynağından arşivlendi. Erişim tarihi: 26 Nisan 2023. Birden fazla yazar-name-list parameters kullanıldı (yardım); Yazar |ad1= eksik |soyadı1= (yardım)
"The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation". European Journal of Paediatric Neurology. 7 (4): 155-9. 2003. doi:10.1016/S1090-3798(03)00060-6. PMID 12865054.
"A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy". Neuromuscular Disorders. 16 (7): 417-26. July 2006. doi:10.1016/j.nmd.2006.03.015. PMC 3260054 $2. PMID 16750368.
"An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients". Neuromuscular Disorders. 17 (9–10): 693-7. October 2007. doi:10.1016/j.nmd.2007.05.009. PMID 17658255.
"Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III". Journal of Child Neurology. 26 (12): 1499-507. December 2011. doi:10.1177/0883073811420294. PMID 21940700.
"Genetic mapping of chronic childhood-onset spinal muscular atrophy to chromosome 5q11.2–13.3". Nature. 344 (6266): 540-1. April 1990. doi:10.1038/344540a0. PMID 2320125.
"Identification and characterization of a spinal muscular atrophy-determining gene". Cell. 80 (1): 155-65. January 1995. doi:10.1016/0092-8674(95)90460-3. PMID 7813012.
"Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy". Science Translational Medicine. 3 (72): 72ra18. March 2011. doi:10.1126/scitranslmed.3001777. PMC 3140425 $2. PMID 21368223.
"Consensus statement for standard of care in spinal muscular atrophy". Journal of Child Neurology. 22 (8): 1027-49. August 2007. doi:10.1177/0883073807305788. PMID 17761659.
"Phenotype modifiers of spinal muscular atrophy: the number of SMN2 gene copies, deletion in the NAIP gene and probably gender influence the course of the disease". Acta Biochimica Polonica. 56 (1): 103-8. 2009. doi:10.18388/abp.2009_2521. PMID 19287802.
"Carrier screening for spinal muscular atrophy (SMA) in 107,611 pregnant women during the period 2005–2009: a prospective population-based cohort study". PLOS ONE. 6 (2): e17067. February 2011. doi:10.1371/journal.pone.0017067. PMC 3045421 $2. PMID 21364876.
"Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens". European Journal of Human Genetics. 20 (1): 27-32. January 2012. doi:10.1038/ejhg.2011.134. PMC 3234503 $2. PMID 21811307.
"Perspectives in genetic counseling for spinal muscular atrophy in the new therapeutic era: early pre-symptomatic intervention and test in minors". European Journal of Human Genetics. 27 (12): 1774-1782. December 2019. doi:10.1038/s41431-019-0415-4. PMC 6871529 $2. PMID 31053787.
"Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening". Journal of Neuromuscular Diseases. 5 (2): 145-158. 29 Mayıs 2018. doi:10.3233/JND-180304. PMC 6004919 $2. PMID 29614695. KB1 bakım: göster-yazarlar (link)
"244th ENMC international workshop: Newborn screening in spinal muscular atrophy May 10-12, 2019, Hoofdorp, The Netherlands". Neuromuscular Disorders. 30 (1): 93-103. January 2020. doi:10.1016/j.nmd.2019.11.002. PMID 31882184.
"Pilot study of population-based newborn screening for spinal muscular atrophy in New York state". Genetics in Medicine. 20 (6): 608-613. June 2018. doi:10.1038/gim.2017.152. PMID 29758563. KB1 bakım: göster-yazarlar (link)
"Carrier frequency of SMN1 related spinal muscular atrophy in north Indian population: The need for population based screening program". American Journal of Medical Genetics Part A. 185 (1): 274-277. October 2020. doi:10.1002/ajmg.a.61918. PMID 33051992.
"Carrier screening for spinal muscular atrophy". Genetics in Medicine. 10 (11): 840-2. November 2008. doi:10.1097/GIM.0b013e318188d069. PMC 3110347 $2. PMID 18941424.
"Spinal muscular atrophy carriers with two SMN1 copies". Brain & Development. 39 (10): 851-860. November 2017. doi:10.1016/j.braindev.2017.06.002. PMID 28676237.
"Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy". New England Journal of Medicine. 377 (18): 1723-32. November 2017. doi:10.1056/NEJMoa1702752. PMID 29091570. 26 Nisan 2023 tarihinde kaynağından arşivlendi. KB1 bakım: göster-yazarlar (link)
Wadman (1 Haziran 2020). "Drug treatment for spinal muscular atrophy types II and III". The Cochrane Database of Systematic Reviews. 1 (1): CD006282. doi:10.1002/14651858.CD006282.pub5. ISSN 1469-493X. PMC 6995983 $2. PMID 32006461.
"Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy". Human Molecular Genetics. 25 (10): 1885-1899. May 2016. doi:10.1093/hmg/ddw062. PMC 5062580 $2. PMID 26931466. KB1 bakım: göster-yazarlar (link)
"Spinal muscular atrophy type 1: A noninvasive respiratory management approach". Chest. 117 (4): 1100-5. April 2000. doi:10.1378/chest.117.4.1100. PMID 10767247.
"Long-term survival in Werdnig-Hoffmann disease". American Journal of Physical Medicine & Rehabilitation. 86 (5): 339-45 quiz 346-8, 379. May 2007. doi:10.1097/PHM.0b013e31804a8505. PMID 17449977.
"Feeding problems and malnutrition in spinal muscular atrophy type II". Neuromuscular Disorders. 18 (5): 389-93. May 2008. doi:10.1016/j.nmd.2008.02.008. PMID 18420410.
"Prevalence and risk factors for feeding and swallowing difficulties in spinal muscular atrophy types II and III". The Journal of Pediatrics. 160 (3): 447-451.e1. March 2012. doi:10.1016/j.jpeds.2011.08.016. PMID 21924737.
"Nutrition and swallowing in pediatric neuromuscular patients". Seminars in Pediatric Neurology. 5 (2): 106-15. June 1998. doi:10.1016/S1071-9091(98)80026-0. PMID 9661244.
"Fatty acid oxidation abnormalities in childhood-onset spinal muscular atrophy: primary or secondary defect(s)?". Pediatric Neurology. 12 (1): 21-30. January 1995. doi:10.1016/0887-8994(94)00100-G. PMID 7748356.
"Abnormal fatty acid metabolism in childhood spinal muscular atrophy". Annals of Neurology. 45 (3): 337-43. March 1999. doi:10.1002/1531-8249(199903)45:3<337::AID-ANA9>3.0.CO;2-U. PMID 10072048.
"Congenital heart disease is a feature of severe infantile spinal muscular atrophy". Journal of Medical Genetics. 45 (10): 635-8. October 2008. doi:10.1136/jmg.2008.057950. PMID 18662980.
"Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice". Human Molecular Genetics. 19 (20): 3906-18. October 2010. doi:10.1093/hmg/ddq330. PMC 2947406 $2. PMID 20693262.
"Cardiac defects contribute to the pathology of spinal muscular atrophy models". Human Molecular Genetics. 19 (20): 4059-71. October 2010. doi:10.1093/hmg/ddq329. PMID 20696672.
"Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery". Human Molecular Genetics. 19 (20): 3895-905. October 2010. doi:10.1093/hmg/ddq300. PMC 2947399 $2. PMID 20639395.
"Intelligence and cognitive function in children and adolescents with spinal muscular atrophy". Neuromuscular Disorders. 12 (2): 130-6. February 2002. doi:10.1016/S0960-8966(01)00274-7. PMID 11738354.
"Cognitive functions in Duchenne muscular dystrophy: a reappraisal and comparison with spinal muscular atrophy". Neuromuscular Disorders. 2 (5–6): 371-8. 1992. doi:10.1016/S0960-8966(06)80008-8. PMID 1300185.
"Behavioural problems in children and adolescents with spinal muscular atrophy and their siblings". Developmental Medicine and Child Neurology. 45 (1): 44-9. January 2003. doi:10.1017/S0012162203000082. PMID 12549754.
"Self-reported quality of life has no correlation with functional status in children and adolescents with spinal muscular atrophy". European Journal of Paediatric Neurology. 15 (1): 36-9. January 2011. doi:10.1016/j.ejpn.2010.07.003. PMID 20800519.
"Laparoscopic Nissen fundoplication during gastrostomy tube placement and noninvasive ventilation may improve survival in type I and severe type II spinal muscular atrophy". Journal of Child Neurology. 22 (6): 727-31. June 2007. doi:10.1177/0883073807304009. PMID 17641258.
"Medical considerations of long-term survival of Werdnig-Hoffmann disease". American Journal of Physical Medicine & Rehabilitation. 86 (5): 349-55. May 2007. doi:10.1097/PHM.0b013e31804b1d66. PMID 17449979.
"The changing natural history of spinal muscular atrophy type 1". Neurology. 69 (20): 1931-6. November 2007. doi:10.1212/01.wnl.0000290830.40544.b9. PMID 17998484.
"Spinal Muscular Atrophy Therapeutics: Where do we Stand?". Neurotherapeutics. 12 (2): 303-16. April 2015. doi:10.1007/s13311-015-0337-y. PMC 4404440 $2. PMID 25631888.
Kletzl (2019). "The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy". Neuromuscular Disorders. Elsevier BV. 29 (1): 21-29. doi:10.1016/j.nmd.2018.10.001. ISSN 0960-8966. PMID 30553700.
"An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA". Gene Therapy. 8 (20): 1532-8. October 2001. doi:10.1038/sj.gt.3301550. PMID 11704813.
"Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients". Human Molecular Genetics. 10 (24): 2841-9. November 2001. doi:10.1093/hmg/10.24.2841. PMID 11734549.
"Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response". Human Molecular Genetics. 24 (22): 6265-77. November 2015. doi:10.1093/hmg/ddv329. PMC 4614699 $2. PMID 26264577.
"Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy". Proceedings of the National Academy of Sciences of the United States of America. 113 (39): 10962-7. September 2016. doi:10.1073/pnas.1605731113. PMC 5047168 $2. PMID 27621445.
"Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells". Journal of Medical Genetics. 45 (1): 29-31. January 2008. doi:10.1136/jmg.2007.051177. PMID 17932121.
"Daily salbutamol in young patients with SMA type II". Neuromuscular Disorders. 18 (7): 536-40. July 2008. doi:10.1016/j.nmd.2008.05.004. PMID 18579379.
"Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design" (PDF). Journal of Medical Genetics. 47 (12): 856-8. December 2010. doi:10.1136/jmg.2010.080366. PMID 20837492. 10 Mart 2022 tarihinde kaynağından arşivlendi (PDF). Erişim tarihi: 26 Nisan 2023.
"P.6.4 Salbutamol tolerability and efficacy in adult type III SMA patients: Results of a multicentric, molecular and clinical, double-blind, placebo-controlled study". Neuromuscular Disorders. 23 (9–10): 771. 2013. doi:10.1016/j.nmd.2013.06.475.
"Treatment of spinal muscular atrophy by sodium butyrate". Proceedings of the National Academy of Sciences of the United States of America. 98 (17): 9808-13. August 2001. doi:10.1073/pnas.171105098. PMC 55534 $2. PMID 11504946.
"Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy". European Journal of Human Genetics. 12 (1): 59-65. January 2004. doi:10.1038/sj.ejhg.5201102. PMID 14560316.
"Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients". European Journal of Human Genetics. 13 (2): 256-9. February 2005. doi:10.1038/sj.ejhg.5201320. PMID 15523494.
"Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy". Neurology. 68 (1): 51-5. January 2007. doi:10.1212/01.wnl.0000249142.82285.d6. PMID 17082463.
"Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy". Human Molecular Genetics. 12 (19): 2481-9. October 2003. doi:10.1093/hmg/ddg256. PMID 12915451.
"Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice". Journal of Molecular Medicine. 86 (11): 1243-54. November 2008. doi:10.1007/s00109-008-0388-1. PMID 18649067.
"SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy". PLOS ONE. 5 (8): e12140. August 2010. doi:10.1371/journal.pone.0012140. PMC 2924376 $2. PMID 20808854.
"SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy". PLOS ONE. 6 (7): e21296. 2011. doi:10.1371/journal.pone.0021296. PMC 3130730 $2. PMID 21754985.
"Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid". BMC Neurology. 11: 36. March 2011. doi:10.1186/1471-2377-11-36. PMC 3078847 $2. PMID 21435220.
"VPA response in SMA is suppressed by the fatty acid translocase CD36". Human Molecular Genetics. 22 (2): 398-407. January 2013. doi:10.1093/hmg/dds437. PMID 23077215.
"Valproic acid blocks excitability in SMA type I mouse motor neurons". Neurobiology of Disease. 36 (3): 477-87. December 2009. doi:10.1016/j.nbd.2009.08.014. PMID 19733665.
"Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis". CNS Drugs. 33 (3): 239-250. March 2019. doi:10.1007/s40263-019-00606-6. PMID 30796634.
"Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells". Annals of Neurology. 58 (2): 194-202. August 2005. doi:10.1002/ana.20548. PMID 16049920.
"Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy". Neurology. 75 (24): 2190-7. December 2010. doi:10.1212/WNL.0b013e3182020332. PMID 21172842.
"Differential regulation of the SMN2 gene by individual HDAC proteins". Biochemical and Biophysical Research Communications. 414 (1): 25-30. October 2011. doi:10.1016/j.bbrc.2011.09.011. PMC 6538936 $2. PMID 21925145.
"The benzamide M344, a novel histone deacetylase inhibitor, significantly increases SMN2 RNA/protein levels in spinal muscular atrophy cells". Human Genetics. 120 (1): 101-10. August 2006. doi:10.1007/s00439-006-0186-1. PMID 16724231.
"LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate". Human Molecular Genetics. 18 (19): 3645-58. October 2009. doi:10.1093/hmg/ddp313. PMID 19584083.
"Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition". Annals of Neurology. 64 (4): 465-70. October 2008. doi:10.1002/ana.21449. PMID 18661558.
"Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy". The Journal of Clinical Investigation. 117 (3): 659-71. March 2007. doi:10.1172/JCI29562. PMC 1797603 $2. PMID 17318264.
"SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy". Human Molecular Genetics. 19 (8): 1492-506. April 2010. doi:10.1093/hmg/ddq023. PMID 20097677.
"Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway". The Journal of Clinical Investigation. 121 (8): 3042-50. August 2011. doi:10.1172/JCI46276. PMC 3148738 $2. PMID 21785216.
"Induction of full-length survival motor neuron by polyphenol botanical compounds". Human Genetics. 122 (6): 635-43. January 2008. doi:10.1007/s00439-007-0441-0. PMID 17962980.
"Histone deacetylase inhibition activity and molecular docking of (e )-resveratrol: its therapeutic potential in spinal muscular atrophy". Chemical Biology & Drug Design. 73 (3): 355-64. March 2009. doi:10.1111/j.1747-0285.2009.00781.x. PMID 19207472.
"Celecoxib increases SMN and survival in a severe spinal muscular atrophy mouse model via p38 pathway activation". Human Molecular Genetics. 22 (17): 3415-24. September 2013. doi:10.1093/hmg/ddt191. PMID 23656793.
"Regulation of SMN protein stability". Molecular and Cellular Biology. 29 (5): 1107-15. March 2009. doi:10.1128/MCB.01262-08. PMC 2643817 $2. PMID 19103745.
"Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts". Human Genetics. 120 (4): 589-601. November 2006. doi:10.1007/s00439-006-0245-7. PMID 16951947.
"Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA". BMC Neuroscience. 10: 142. November 2009. doi:10.1186/1471-2202-10-142. PMC 2789732 $2. PMID 19948047.
"Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism". Chemistry & Biology. 11 (11): 1489-93. November 2004. doi:10.1016/j.chembiol.2004.08.024. PMC 3160629 $2. PMID 15555999.
"Efficacy of thyrotropin-releasing hormone in the treatment of spinal muscular atrophy". Journal of Child Neurology. 9 (3): 287-9. July 1994. doi:10.1177/088307389400900313. PMID 7930408.
"A study of thyrotropin-releasing hormone for the treatment of spinal muscular atrophy: a preliminary report". American Journal of Physical Medicine & Rehabilitation. 79 (5): 435-40. 2000. doi:10.1097/00002060-200009000-00005. PMID 10994885.
"Oral administration of the thyrotropin-releasing hormone (TRH) analogue, taltireline hydrate, in spinal muscular atrophy". Journal of Child Neurology. 24 (8): 1010-2. August 2009. doi:10.1177/0883073809333535. PMID 19666885.
"Drug treatment for spinal muscular atrophy type I". Cochrane Database of Systematic Reviews. John Wiley & Sons, Ltd (12): CD006281. 7 Aralık 2011. doi:10.1002/14651858.cd006281.pub3. PMID 22161399.
"Riluzole attenuates spinal muscular atrophy disease progression in a mouse model". Muscle & Nerve. 28 (4): 432-7. October 2003. doi:10.1002/mus.10455. PMID 14506714.
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dailymed.nlm.nih.gov

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ninds.nih.gov

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rarediseases.org

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sma-screening-alliance.org

"SMA Newborn Screening Alliance – SMA: Test at birth, save a life" (İngilizce). 11 Nisan 2021 tarihinde kaynağından arşivlendi. Erişim tarihi: 5 Şubat 2023.

smanewstoday.com

"SMA Added to List of Recommended Screenings for Disease Given to..." SMA News Today (İngilizce). 16 Temmuz 2018. 12 Mayıs 2019 tarihinde kaynağından arşivlendi. Erişim tarihi: 4 Mayıs 2020. Yazar |ad1= eksik |soyadı1= (yardım)
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"Pediatric Phase 2/3 Trial to Test Anti-myostatin Antibody with Evrysdi" (İngilizce). 26 Ekim 2021 tarihinde kaynağından arşivlendi. Erişim tarihi: 23 Ocak 2022. Yazar |ad1= eksik |soyadı1= (yardım)

treat-nmd.eu

"National registries for DMD, SMA and DM". 22 Ocak 2011 tarihinde kaynağından arşivlendi.

ucl.ac.uk

discovery.ucl.ac.uk

"Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy". New England Journal of Medicine. 377 (18): 1723-32. November 2017. doi:10.1056/NEJMoa1702752. PMID 29091570. 26 Nisan 2023 tarihinde kaynağından arşivlendi. KB1 bakım: göster-yazarlar (link)

unimi.it

air.unimi.it

"Beta-lactam antibiotic offers neuroprotection in a spinal muscular atrophy model by multiple mechanisms" (PDF). Experimental Neurology. 229 (2): 214-25. June 2011. doi:10.1016/j.expneurol.2011.01.017. PMID 21295027. 29 Ağustos 2021 tarihinde kaynağından arşivlendi (PDF). Erişim tarihi: 26 Nisan 2023.

uptodate.com

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web.archive.org

"Spinal muscular atrophy". Genetic and Rare Diseases Information Center (GARD) – an NCATS Program. 23 Ocak 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 27 Mayıs 2019.
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"Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy". New England Journal of Medicine. 377 (18): 1723-32. November 2017. doi:10.1056/NEJMoa1702752. PMID 29091570. 26 Nisan 2023 tarihinde kaynağından arşivlendi. KB1 bakım: göster-yazarlar (link)
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Maria Joao Almeida (8 Eylül 2016). "RG7916". BioNews Services. 13 Eylül 2016 tarihinde kaynağından arşivlendi. Erişim tarihi: 8 Ekim 2017.
"Spinal Muscular Atrophy". uptodate.com. November 2017. 6 Aralık 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 1 Aralık 2017. Yazar |ad1= eksik |soyadı1= (yardım)
"SMA CARE SERIES – Musculoskeletal System" (PDF). www.curesma.org. Yaz 2017. 19 Şubat 2018 tarihinde kaynağından (PDF) arşivlendi. Erişim tarihi: 7 Aralık 2017. Yazar |ad1= eksik |soyadı1= (yardım)
"Novartis Releases Update on LMI070 (Branaplam) Clinical Trial". CureSMA. 25 Kasım 2017 tarihinde kaynağından arşivlendi. Erişim tarihi: 7 Ekim 2017.
"Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design" (PDF). Journal of Medical Genetics. 47 (12): 856-8. December 2010. doi:10.1136/jmg.2010.080366. PMID 20837492. 10 Mart 2022 tarihinde kaynağından arşivlendi (PDF). Erişim tarihi: 26 Nisan 2023.
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"Beta-lactam antibiotic offers neuroprotection in a spinal muscular atrophy model by multiple mechanisms" (PDF). Experimental Neurology. 229 (2): 214-25. June 2011. doi:10.1016/j.expneurol.2011.01.017. PMID 21295027. 29 Ağustos 2021 tarihinde kaynağından arşivlendi (PDF). Erişim tarihi: 26 Nisan 2023.
"CK-2127107". 16 Ekim 2014 tarihinde kaynağından arşivlendi.
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worldcat.org

Verhaart (July 2017). "A multi-source approach to determine SMA incidence and research ready population". Journal of Neurology (İngilizce). 264 (7): 1465-1473. doi:10.1007/s00415-017-8549-1. ISSN 0340-5354. PMC 5502065 $2. PMID 28634652.
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Wadman (1 Haziran 2020). "Drug treatment for spinal muscular atrophy types II and III". The Cochrane Database of Systematic Reviews. 1 (1): CD006282. doi:10.1002/14651858.CD006282.pub5. ISSN 1469-493X. PMC 6995983 $2. PMID 32006461.
"$2.1m Novartis gene therapy to become world's most expensive drug". The Guardian. Reuters. 25 Mayıs 2019. ISSN 0261-3077.
Kletzl (2019). "The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy". Neuromuscular Disorders. Elsevier BV. 29 (1): 21-29. doi:10.1016/j.nmd.2018.10.001. ISSN 0960-8966. PMID 30553700.

wsj.com

"Surprise Drug Approval Is Holiday Gift for Biogen". The Wall Street Journal. 27 Aralık 2016. ISSN 0099-9660. 8 Kasım 2020 tarihinde kaynağından arşivlendi. Erişim tarihi: 27 Aralık 2016. Birden fazla yazar-name-list parameters kullanıldı (yardım); Yazar |ad1= eksik |soyadı1= (yardım)