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@ARTICLE{Klopstock:154280,
      author       = {Klopstock, Thomas and Videnovic, Aleksandar and Bischoff,
                      Almut Turid and Bonnet, Cecilia and Cif, Laura and Comella,
                      Cynthia and Correa-Vela, Marta and Escolar, Maria L and
                      Fraser, Jamie L and Gonzalez, Victoria and Hermanowicz, Neal
                      and Jech, Robert and Jinnah, Hyder A and Kmiec, Tomasz and
                      Lang, Anthony and Martí, Maria J and Mercimek-Andrews,
                      Saadet and Monduy, Migvis and Nimmo, Graeme A M and
                      Perez-Dueñas, Belen and Pfeiffer, Helle Cecilie Viekilde
                      and Planellas, Lluis and Roze, Emmanuel and Thakur, Nivedita
                      and Tochen, Laura and Vanegas-Arroyave, Nora and Zorzi,
                      Giovanna and Burns, Colleen and Greblikas, Feriandas},
      title        = {{F}osmetpantotenate {R}andomized {C}ontrolled {T}rial in
                      {P}antothenate {K}inase-{A}ssociated {N}eurodegeneration.},
      journal      = {Movement disorders},
      volume       = {36},
      number       = {6},
      issn         = {1531-8257},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2021-00134},
      pages        = {1342 - 1352},
      year         = {2020},
      note         = {ISSN 1531-8257 not unique: **3 hits**.},
      abstract     = {Pantothenate kinase-associated neurodegeneration (PKAN)
                      currently has no approved treatments.The Fosmetpantotenate
                      Replacement Therapy pivotal trial examined whether treatment
                      with fosmetpantotenate improves PKAN symptoms and stabilizes
                      disease progression.This randomized, double-blind,
                      placebo-controlled, multicenter study evaluated
                      fosmetpantotenate, 300 mg oral dose three times daily,
                      versus placebo over a 24-week double-blind period. Patients
                      with pathogenic variants of PANK2, aged 6 to 65 years, with
                      a score ≥6 on the PKAN-Activities of Daily Living
                      (PKAN-ADL) scale were enrolled. Patients were randomized to
                      active (fosmetpantotenate) or placebo treatment, stratified
                      by weight and age. The primary efficacy endpoint was change
                      from baseline at week 24 in PKAN-ADL.Between July 23, 2017,
                      and December 18, 2018, 84 patients were randomized
                      (fosmetpantotenate: n = 41; placebo: n = 43); all 84
                      patients were included in the analyses. Six patients in the
                      placebo group discontinued treatment; two had worsening
                      dystonia, two had poor compliance, and two died of
                      PKAN-related complications (aspiration during feeding and
                      disease progression with respiratory failure, respectively).
                      Fosmetpantotenate and placebo group PKAN-ADL mean (standard
                      deviation) scores were 28.2 (11.4) and 27.4 (11.5) at
                      baseline, respectively, and were 26.9 (12.5) and 24.5 (11.8)
                      at week 24, respectively. The difference in least square
                      mean $(95\%$ confidence interval) at week 24 between
                      fosmetpantotenate and placebo was -0.09 (-1.69 to 1.51; P =
                      0.9115). The overall incidence of treatment-emergent serious
                      adverse events was similar in the fosmetpantotenate (8/41;
                      $19.5\%)$ and placebo (6/43; $14.0\%)$ groups.Treatment with
                      fosmetpantotenate was safe but did not improve function
                      assessed by the PKAN-ADL in patients with PKAN. © 2020 The
                      Authors. Movement Disorders published by Wiley Periodicals
                      LLC on behalf of International Parkinson and Movement
                      Disorder Society.},
      keywords     = {Activities of Daily Living / Double-Blind Method / Humans /
                      Pantothenate Kinase-Associated Neurodegeneration: drug
                      therapy / Pantothenate Kinase-Associated Neurodegeneration:
                      genetics / Pantothenic Acid: analogs $\&$ derivatives /
                      fosmetpantotenate (Other) / pantothenate kinase-associated
                      neurodegeneration (Other) / randomized controlled trial
                      (Other) / treatment (Other)},
      cin          = {Clinical Dementia Research München},
      ddc          = {610},
      cid          = {I:(DE-2719)1111016},
      pnm          = {344 - Clinical and Health Care Research (POF3-344)},
      pid          = {G:(DE-HGF)POF3-344},
      typ          = {PUB:(DE-HGF)16},
      pmc          = {pmc:PMC8246547},
      pubmed       = {pmid:33200489},
      doi          = {10.1002/mds.28392},
      url          = {https://pub.dzne.de/record/154280},
}