التفاصيل البيبلوغرافية
| العنوان: |
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. |
| المؤلفون: |
Finkel, Richard S, Mercuri, Eugenio, Darras, Basil T, Connolly, Anne M, Kuntz, Nancy L, Kirschner, Janbernd, Chiriboga, Claudia A, Saito, Kayoko, Deconinck, Nicolas, Tizzano, Eduardo, Topaloglu, Haluk, Tulinius, Már, Montes, Jacqueline, Glanzman, Allan M, Bishop, Kathie, Zhong, Z John, Gheuens, Sarah, Bennett, C Frank, Schneider, Eugene, Farwell, Wildon, De Vivo, Darryl C., ENDEAR Study Group |
| المصدر: |
The New England journal of medicine, 377 (18 |
| بيانات النشر: |
2017 |
| نوع الوثيقة: |
Electronic Resource |
| مستخلص: |
Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein.
info:eu-repo/semantics/published |
| مصطلحات الفهرس: |
Sciences bio-médicales et agricoles, Age of Onset, Disease-Free Survival, Double-Blind Method, Female, Humans, Infant, Injections, Spinal, Male, Motor Skills, Oligonucleotides -- adverse effects -- therapeutic use, Oligonucleotides, Antisense -- adverse effects -- therapeutic use, RNA, Messenger -- drug effects -- metabolism, Respiration, Artificial, Spinal Muscular Atrophies of Childhood -- drug therapy -- genetics -- mortality -- physiopathology, Survival Analysis, Survival of Motor Neuron 2 Protein -- genetics -- metabolism, info:eu-repo/semantics/article, info:ulb-repo/semantics/articlePeerReview, info:ulb-repo/semantics/openurl/article |
| URL: |
http://hdl.handle.net/2013/ULB-DIPOT:oai:dipot.ulb.ac.be:2013/262825
http://worldcat.org/search?q=on:EQY+http://difusion-oai.ulb.ac.be/oai/request+DCG_ENTIRE_REPOSITORY+CNTCOLL |
| الإتاحة: |
Open access content. Open access content |
| ملاحظة: |
No full-text files
English |
| أرقام أخرى: |
EQY oai:dipot.ulb.ac.be:2013/262825
uri/info:doi/10.1056/NEJMoa1702752
uri/info:pmid/29091570
1021240106 |
| المصدر المساهم: |
UNIVERSITE LIBRE DE BRUXELLES
From OAIster®, provided by the OCLC Cooperative. |
| رقم الأكسشن: |
edsoai.on1021240106 |
| قاعدة البيانات: |
OAIster |
