Volume 2; Issue 11

Expert Opinion on Orphan Drugs

Volume 2; Issue 11
2

Genethon: patient-empowered research

Year:
2014
Language:
english
File:
PDF, 303 KB
english, 2014
3

The emergence of gene therapy for rare diseases

Year:
2014
Language:
english
File:
PDF, 391 KB
english, 2014
4

Resources

Year:
2014
Language:
english
File:
PDF, 90 KB
english, 2014
7

Ivacaftor therapy for cystic fibrosis

Year:
2014
Language:
english
File:
PDF, 456 KB
english, 2014
8

Bortezomib for the treatment of mantle cell lymphoma

Year:
2014
Language:
english
File:
PDF, 319 KB
english, 2014
10

Findacure: empowering patient groups for drug development

Year:
2014
Language:
english
File:
PDF, 206 KB
english, 2014
14

The diagnostic gap – an expert opinion

Year:
2014
Language:
english
File:
PDF, 349 KB
english, 2014
15

A hidden priority: the paradox of rarity (NORD perspective)

Year:
2014
Language:
english
File:
PDF, 331 KB
english, 2014
16

How close are we to a cure for Duchenne muscular dystrophy?

Year:
2014
Language:
english
File:
PDF, 447 KB
english, 2014
17

Adventures of an advocate: Annette De Bow’s big journeys

Year:
2014
Language:
english
File:
PDF, 405 KB
english, 2014
18

A US perspective on newborn screening: a powerful tool for prevention

Year:
2014
Language:
english
File:
PDF, 382 KB
english, 2014
19

A hidden priority: the paradox of rarity (EURORDIS perspective)

Year:
2014
Language:
english
File:
PDF, 372 KB
english, 2014
21

Knowing more about Noonan, by Samantha Scheer

Year:
2014
File:
PDF, 433 KB
2014
22

Stiff person syndrome, by Liz Blows

Year:
2014
File:
PDF, 429 KB
2014