scholarly article | Q13442814 |
editorial | Q871232 |
P6179 | Dimensions Publication ID | 1091497563 |
P356 | DOI | 10.1038/GT.2017.79 |
P698 | PubMed publication ID | 28880019 |
P50 | author | Steven Simoens | Q87433253 |
P2093 | author name string | I Huys | |
P2860 | cites work | Spinal muscular atrophy. | Q33345749 |
Pricing and reimbursement of orphan drugs: the need for more transparency | Q34193502 | ||
Structure and organization of the human survival motor neurone (SMN) gene | Q34400462 | ||
Big win possible for Ionis/Biogen antisense drug in muscular atrophy | Q34542348 | ||
Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? | Q36639084 | ||
Advanced therapy medicinal products: current and future perspectives | Q36840842 | ||
Rare diseases, orphan drugs and their regulation: questions and misconceptions | Q43814369 | ||
A pilot study of multicriteria decision analysis for valuing orphan medicines. | Q48119113 | ||
Resveratrol suppressed seizures by attenuating IL-1β, IL1-Ra, IL-6, and TNF-α in the hippocampus and cortex of kindled mice | Q48214403 | ||
Classification of spinal muscular atrophies | Q72423037 | ||
An Update on Clinical And Economic Evidence Requirements for Advanced-Therapy Medicinal Products in Europe | Q89240473 | ||
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy | Q89262117 | ||
Fresh from the biotech pipeline-2016 | Q89947270 | ||
P433 | issue | 9 | |
P921 | main subject | muscular atrophy | Q2844600 |
intellectual property | Q131257 | ||
spinal muscular atrophy | Q580290 | ||
intellectual property right | Q108855835 | ||
P304 | page(s) | 539-541 | |
P577 | publication date | 2017-09-07 | |
P1433 | published in | Gene Therapy | Q15763095 |
P1476 | title | Market access of Spinraza (Nusinersen) for spinal muscular atrophy: intellectual property rights, pricing, value and coverage considerations | |
P478 | volume | 24 |
Q60046512 | Barriers for Access to New Medicines: Searching for the Balance Between Rising Costs and Limited Budgets |
Q49922954 | Genetic therapies for spinal muscular atrophy type 1. |
Q99417440 | RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges |
Q90309160 | RNA therapy: Are we using the right molecules? |
Q91975720 | The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach |
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