Can We Generalize Key Principles in the Care of Rare Diseases? The Case for Adrenoleukodystrophy

Affiliations

¹ From the Division of Pediatric Neurology (M.D.C.), University of Michigan Medical School, Ann Arbor, MI; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology (P.B.K.), University of Minnesota Medical School, Minneapolis, MN; and Institute for Translational Neuroscience (P.B.K.), University of Minnesota, Minneapolis, MN.
² From the Division of Pediatric Neurology (M.D.C.), University of Michigan Medical School, Ann Arbor, MI; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology (P.B.K.), University of Minnesota Medical School, Minneapolis, MN; and Institute for Translational Neuroscience (P.B.K.), University of Minnesota, Minneapolis, MN. pkang@umn.edu.

Editorial

Martha D Carlson et al. Neurology. 2022.

. 2022 Nov 22;99(21):929-930.

doi: 10.1212/WNL.0000000000201400. Epub 2022 Sep 29.

¹ From the Division of Pediatric Neurology (M.D.C.), University of Michigan Medical School, Ann Arbor, MI; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology (P.B.K.), University of Minnesota Medical School, Minneapolis, MN; and Institute for Translational Neuroscience (P.B.K.), University of Minnesota, Minneapolis, MN.
² From the Division of Pediatric Neurology (M.D.C.), University of Michigan Medical School, Ann Arbor, MI; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology (P.B.K.), University of Minnesota Medical School, Minneapolis, MN; and Institute for Translational Neuroscience (P.B.K.), University of Minnesota, Minneapolis, MN. pkang@umn.edu.

No abstract available

Comment on

International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach.
Engelen M, van Ballegoij WJC, Mallack EJ, Van Haren KP, Köhler W, Salsano E, van Trotsenburg ASP, Mochel F, Sevin C, Regelmann MO, Tritos NA, Halper A, Lachmann RH, Davison J, Raymond GV, Lund TC, Orchard PJ, Kuehl JS, Lindemans CA, Caruso P, Turk BR, Moser AB, Vaz FM, Ferdinandusse S, Kemp S, Fatemi A, Eichler FS, Huffnagel IC. Engelen M, et al. Neurology. 2022 Nov 22;99(21):940-951. doi: 10.1212/WNL.0000000000201374. Epub 2022 Sep 29. Neurology. 2022. PMID: 36175155 Free PMC article.