Victor McKusick and his role in the founding of the European School of Genetic Medicine

Author(s):  
Giovanni Romeo ◽  
Martin Bobrow ◽  
Malcolm Ferguson‐Smith ◽  
Andrea Ballabio
2019 ◽  
Vol 10 (9) ◽  
pp. 555-561
Author(s):  
Louise Rosenmayr-Templeton

This industry update features a round-up of pharmaceutical news in May 2019 based on press releases and websites. The month was characterized by the achievement of significant milestones in gene therapy. The biggest of these was the US FDA’s approval of Zolgensma®. This medicine sums up the promise and price of genetic medicine. On one hand the clinical results show Zolgensma can dramatically improve the prognosis for infants with spinal muscular atrophy after just one administration, while on the other, it has been priced at around US$2.1 million. With more such therapies likely to reach the market, the debate on Zolgensma goes beyond cost, to overall affordability, the true meaning of cost–effectiveness and how to reward companies for effective, innovative medicines.


1988 ◽  
Vol 11 (4) ◽  
pp. 259???261
Author(s):  
A. A. M. De Haas

Cancer ◽  
2009 ◽  
Vol 115 (S13) ◽  
pp. 3035-3038 ◽  
Author(s):  
Riccardo Valdagni ◽  
Peter T. Scardino ◽  
Louis Denis

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