Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases

RESEARCH

Factors associated with refractory autoimmune necrotizing myopathy with anti-signal recognition particle antibodies

Yawen Zhao, Wei Zhang, Yilin Liu, Zhaoxia Wang and Yun Yuan

Orphanet Journal of Rare Diseases 2020, 15:181 | Published on: 8 July 2020

Full Text | PDF

RESEARCH

Exome sequencing for diagnosis of congenital hemolytic anemia

Lamisse Mansour-Hendili, Abdelrazak Aissat, Bouchra Badaoui, Mehdi Sakka, Christine Gameiro, Valérie Ortonne, Orianne Wagner-Ballon, Serge Pissard, Véronique Picard, Khaldoun Ghazal, Michel Bahuau, Corinne Guitton, Ziad Mansour, Mylène Duplan, Arnaud Petit, Nathalie Costedoat-Chalumeau…

Orphanet Journal of Rare Diseases 2020, 15:180 | Published on: 8 July 2020

Full Text | PDF

RESEARCH

A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

Teresa Coelho, David Adams, Isabel Conceição, Márcia Waddington-Cruz, Hartmut H. Schmidt, Juan Buades, Josep Campistol, John L. Berk, Michael Polydefkis, Jing Jing Wang, Jihong Chen, Marianne T. Sweetser, Jared Gollob and Ole B. Suhr

Orphanet Journal of Rare Diseases 2020, 15:179 | Published on: 8 July 2020

Full Text | PDF

Follow BMC on:

For further information or enquiries please use our contact page details.

BMC respects your privacy and does not disclose, sell or rent your personal information to any non-affliated third parties with your consent. Privacy policy.

BioMed Central Ltd

The Campus, 4 Crinan Street,

London, N1 9XW,

United Kingdom