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Drisapersen
Opportunities and Challenges for Antisense Oligonucleotide Therapies
The Use of Ataluren in the Effective Management of Duchenne Muscular Dystrophy
Are Speci C Symptoms and Signs That Will Enable the Clinician to Recognise Disease Patterns That Will Narrow Down the Differenti
Eteplirsen
PRAC Draft Agenda of Meeting 14-17 March 2016
Annexes to the Annual Report of the European Medicines Agency 2014
Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
Curriculum Vitae
213026Orig1s000
Systematic Mining of Gene Co-Expression Network Suggesting a New Drug Repositioning for the Effective Treatment of Duchenne Muscular Dystrophy
What Is the Level of Dystrophin Expression Required for Effective
Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy
Deoxyuridine Nucleotides Induce Exon-Skipping I
Der Einfluss Der VO (EG) 1901/2006 Auf Den Off-Label Use in Der Neonatologie“
Landscape Review and Evidence Map of Gene Therapy, Part 2
Eteplirsen NDA 206488
Stembook 2018.Pdf
FDA Briefing Document
Top View
Sustainability of the European Regulatory System for Medicinal Products
Pediatric Pharmacotherapy
(INN) for Biological and Biotechnological Substances
RNA-Targeting Splicing Modifiers: Drug Development and Screening Assays
AHRQ Healthcare Horizon Scanning System – Status Update Horizon
AHRQ Healthcare Horizon Scanning System – Status Update
Antisense Oligonucleotides: Treatment Strategies and Cellular Internalization Colton M
FSH Society Grants Awarded August 2015 Cycle
Alrohaif, H 2018.Pdf
209531Orig1s000
Neurology Care, Diagnostics, and Emerging Therapies of the Patient
Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders Flavien Bizot, Adeline Vulin, Aurélie Goyenvalle
Drug Against Duchenne Muscular Dystrophy Following Appeal
Extract from Clinical Evaluation Nusinersen (As Heptadecasodium)
Fontelonga Unr 0139D 12728.Pdf
Effectiveness of Pharmacological Treatments In
ATC-Index Mit DDD-Angaben Für Deutschland Im Jahre 2019
Current Translational Research and Murine Models for Duchenne Muscular Dystrophy
Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value
Fresh from the Biotech Pipeline—2016
Exondys, Through the Centralised Procedure Falling Within the Article 3(1) and Point 4 of Annex of Regulation (EC) No 726/2004
Therapeutic Strategies for Duchenne Muscular Dystrophy: an Update
Duchenne Muscular Dystrophy Genetic Therapies Approved By: UM Pharmacy Subcommittee
The Use of Stems in the Selection of International Nonproprietary Names (INN) for Pharmaceutical Substances
THESE Nivine SROUR
[Ehdsi V3.0.0-RC1] Ehealth DSI – Master Value Set Catalogue
A New Era in the Management of Neuromuscular Disorders in Children and Adolescents
Pipeline Highlights 2016: Focus on Duchenne Muscular Dystrophy
Comparison of Results from the STRIDE Registry and CINRG DMD Natural History Study
Tenofovir Alafenamide
Exon Skipping for Duchenne Muscular Dystrophy
Molekulare Therapien Bei Neuromuskulären Erkrankungen Im Kindesalter – Große Hoffnungen Und Unbekannte Risiken
Realizing the Potential of Rna-Based Technology
Exondys 51™ (Eteplirsen)
Pbr Excluded Drugs List
Advisory Committee Briefing Materials: Available for Public Release
FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides