Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases RESEARCH Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study Frédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, Paola Ramirez-Garcia, Julien Durigneux, Christine Barnerias, Arnaud Isapof, Jean-Marie Cuisset, Claude Cances, Christian Richelme, Carole Vuillerot, Vincent Laugel, Juliette Ropars, Cécilia Altuzarra, Caroline Espil-Taris, Ulrike Walther-Louvier… Orphanet Journal of Rare Diseases 2020, 15:148 | Published on: 12 June 2020 Full Text | PDF RESEARCH Juvenile xanthogranuloma as a new type of skin lesions in tuberous sclerosis complex Qian Lu, Xiu-Yu Shi, Yang-Yang Wang, Meng-Na Zhang, Wen-Ze Wang, Jing Wang, Qiu-Hong Wang, Hui-Min Chen and Li-Ping Zou Orphanet Journal of Rare Diseases 2020, 15:147 | Published on: 12 June 2020 Full Text | PDF RESEARCH Costs of illness in amyotrophic lateral sclerosis (ALS): a cross-sectional survey in Germany Erik Schönfelder, Alma Osmanovic, Lars Hendrik Müschen, Susanne Petri and Olivia Schreiber-Katz Orphanet Journal of Rare Diseases 2020, 15:149 | Published on: 12 June 2020 Full Text | PDF

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