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The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. [electronic resource] by
- Wang, Raymond Y
- da Silva Franco, José Francisco
- López-Valdez, Jaime
- Martins, Esmeralda
- Sutton, Vernon Reid
- Whitley, Chester B
- Zhang, Lin
- Cimms, Tricia
- Marsden, Deborah
- Jurecka, Agnieszka
- Harmatz, Paul
Producer: 20201123
In:
Molecular genetics and metabolism vol. 129
Availability: No items available.
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Bi-allelic CSF1R Mutations Cause Skeletal Dysplasia of Dysosteosclerosis-Pyle Disease Spectrum and Degenerative Encephalopathy with Brain Malformation. [electronic resource] by
- Guo, Long
- Bertola, Débora Romeo
- Takanohashi, Asako
- Saito, Asuka
- Segawa, Yuko
- Yokota, Takanori
- Ishibashi, Satoru
- Nishida, Yoichiro
- Yamamoto, Guilherme Lopes
- Franco, José Francisco da Silva
- Honjo, Rachel Sayuri
- Kim, Chong Ae
- Musso, Camila Manso
- Timmons, Margaret
- Pizzino, Amy
- Taft, Ryan J
- Lajoie, Bryan
- Knight, Melanie A
- Fischbeck, Kenneth H
- Singleton, Andrew B
- Ferreira, Carlos R
- Wang, Zheng
- Yan, Li
- Garbern, James Y
- Simsek-Kiper, Pelin O
- Ohashi, Hirofumi
- Robey, Pamela G
- Boyde, Alan
- Matsumoto, Naomichi
- Miyake, Noriko
- Spranger, Jürgen
- Schiffmann, Raphael
- Vanderver, Adeline
- Nishimura, Gen
- Passos-Bueno, Maria Rita Dos Santos
- Simons, Cas
- Ishikawa, Kinya
- Ikegawa, Shiro
Producer: 20200206
In:
American journal of human genetics vol. 104
Availability: No items available.
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