Recommendations for patient screening in ultra-rare inherited metabolic diseases: What have we learned from Niemann-Pick disease type C?
- Sobrido, M.-J.
- Bauer, P.
- De Koning, T.
- Klopstock, T.
- Nadjar, Y.
- Patterson, M.C.
- Synofzik, M.
- Hendriksz, C.J.
Revista:
Orphanet Journal of Rare Diseases
ISSN: 1750-1172
Ano de publicación: 2019
Volume: 14
Número: 1
Tipo: Revisión
