Volume 6; Issue 3

Expert Opinion on Orphan Drugs

Volume 6; Issue 3

1

Developing role of eltrombopag in the treatment of aplastic anemia

Scheinberg, Phillip

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 711 KB

english, 2018

2

Human plasma-derived C1 esterase inhibitor for on-demand or prophylaxis treatment of patients with hereditary angioedema: intravenous and subcutaneous formulations

Bernstein, Jonathan A.

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 1.15 MB

english, 2018

3

Gene therapy for the treatment of X-linked retinitis pigmentosa

Martinez-Fernandez De La Camara, Cristina, Nanda, Anika, Salvetti, Anna Paola, Fischer, M. Dominik, MacLaren, Robert E.

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 1.40 MB

english, 2018

4

Advances in the understanding of hereditary ataxia – implications for future patients

Zeitlberger, Anna, Ging, Heather, Nethisinghe, Suran, Giunti, Paola

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 1.35 MB

english, 2018

5

Clinical trial design for Friedreich ataxia - Where are we now and what do we need?

Rummey, Christian, Kichula, Elizabeth, Lynch, David R.

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 1.35 MB

english, 2018

6

The potential of utrophin modulators for the treatment of Duchenne muscular dystrophy

Guiraud, Simon, Roblin, David, Kay, Davies. E.

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 1.77 MB

english, 2018

7

Lessons from chylomicron retention disease: a potential new approach for the treatment of hypercholesterolemia?

Journal:

Expert Opinion on Orphan Drugs

Année:

2018

Langue:

english

Fichier:

PDF, 571 KB

english, 2018