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101.
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102.
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SMA screening system using dried blood spots on filter paper: application of COP-PCR to the SMN1 deletion test. [electronic resource] by
- Kato, Nozomu
- Sa'Adah, Nihayatus
- Ar Rochmah, Mawaddah
- Harahap, Nur Imma Fatimah
- Nurputra, Dian Kesumapramudya
- Sato, Hideyuki
- Sadewa, Ahmad Hamim
- Astuti, Indwiani
- Haryana, Sofia Mubarika
- Saito, Toshio
- Saito, Kayoko
- Nishimura, Noriyuki
- Nishio, Hisahide
- Takeuchi, Atsuko
Producer: 20160317
In:
The Kobe journal of medical sciences vol. 60
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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment. [electronic resource] by
- Ramos, Daniel M
- d'Ydewalle, Constantin
- Gabbeta, Vijayalakshmi
- Dakka, Amal
- Klein, Stephanie K
- Norris, Daniel A
- Matson, John
- Taylor, Shannon J
- Zaworski, Phillip G
- Prior, Thomas W
- Snyder, Pamela J
- Valdivia, David
- Hatem, Christine L
- Waters, Ian
- Gupte, Nikhil
- Swoboda, Kathryn J
- Rigo, Frank
- Bennett, C Frank
- Naryshkin, Nikolai
- Paushkin, Sergey
- Crawford, Thomas O
- Sumner, Charlotte J
Producer: 20200608
In:
The Journal of clinical investigation vol. 129
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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. [electronic resource] by
- De Vivo, Darryl C
- Bertini, Enrico
- Swoboda, Kathryn J
- Hwu, Wuh-Liang
- Crawford, Thomas O
- Finkel, Richard S
- Kirschner, Janbernd
- Kuntz, Nancy L
- Parsons, Julie A
- Ryan, Monique M
- Butterfield, Russell J
- Topaloglu, Haluk
- Ben-Omran, Tawfeg
- Sansone, Valeria A
- Jong, Yuh-Jyh
- Shu, Francy
- Staropoli, John F
- Kerr, Douglas
- Sandrock, Alfred W
- Stebbins, Christopher
- Petrillo, Marco
- Braley, Gabriel
- Johnson, Kristina
- Foster, Richard
- Gheuens, Sarah
- Bhan, Ishir
- Reyna, Sandra P
- Fradette, Stephanie
- Farwell, Wildon
Producer: 20200831
In:
Neuromuscular disorders : NMD vol. 29
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108.
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A SMN2 Splicing Modifier Rescues the Disease Phenotypes in an In Vitro Human Spinal Muscular Atrophy Model. [electronic resource] by
- Son, Ye Seul
- Choi, Kwangman
- Lee, Hana
- Kwon, Ohman
- Jung, Kwang Bo
- Cho, Sunwha
- Baek, Jiyeon
- Son, Bora
- Kang, Sung-Min
- Kang, Mingu
- Yoon, Jihee
- Shen, Haihong
- Lee, Sangku
- Oh, Jung-Hwa
- Lee, Hyang-Ae
- Lee, Mi-Ok
- Cho, Hyun-Soo
- Jung, Cho-Rok
- Kim, Janghwan
- Cho, Sungchan
- Son, Mi-Young
Producer: 20200305
In:
Stem cells and development vol. 28
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118.
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Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice. [electronic resource] by
- Rashnonejad, Afrooz
- Amini Chermahini, Gholamhossein
- Gündüz, Cumhur
- Onay, Hüseyin
- Aykut, Ayça
- Durmaz, Burak
- Baka, Meral
- Su, Qin
- Gao, Guangping
- Özkınay, Ferda
Producer: 20200626
In:
Molecular therapy : the journal of the American Society of Gene Therapy vol. 27
Availability: No items available.
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