These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

130 related articles for article (PubMed ID: 34250946)

  • 1. Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy.
    Lloyd A; Aggio D; Slocomb TL; Lee J; Beggs AH; Bilder DA
    J Neuromuscul Dis; 2021; 8(6):1047-1061. PubMed ID: 34250946
    [TBL] [Abstract][Full Text] [Related]  

  • 2. An algorithm for discontinuing mechanical ventilation in boys with x-linked myotubular myopathy after positive response to gene therapy: the ASPIRO experience.
    Graham RJ; Amin R; Demirel N; Edel L; Lilien C; MacBean V; Rafferty GF; Sawnani H; Schön C; Smith BK; Syed F; Sarazen M; Prasad S; Rico S; Perez GF
    Respir Res; 2024 Sep; 25(1):342. PubMed ID: 39285418
    [TBL] [Abstract][Full Text] [Related]  

  • 3. INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
    Dowling JJ; Müller-Felber W; Smith BK; Bönnemann CG; Kuntz NL; Muntoni F; Servais L; Alfano LN; Beggs AH; Bilder DA; Blaschek A; Duong T; Graham RJ; Jain M; Lawlor MW; Lee J; Coats J; Lilien C; Lowes LP; MacBean V; Neuhaus S; Noursalehi M; Pitts T; Finlay C; Christensen S; Rafferty G; Seferian AM; Tsuchiya E; James ES; Miller W; Sepulveda B; Vila MC; Prasad S; Rico S; Shieh PB;
    J Neuromuscul Dis; 2022; 9(4):503-516. PubMed ID: 35694931
    [TBL] [Abstract][Full Text] [Related]  

  • 4. A healthcare claims analysis to identify and characterize patients with suspected X-Linked Myotubular Myopathy (XLMTM) in the Brazilian Healthcare System.
    Souza PVS; Haselkorn T; Baima J; Oliveira RW; Hernández F; Birck MG; França MC
    Orphanet J Rare Dis; 2024 May; 19(1):188. PubMed ID: 38715109
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Health state utilities associated with caring for an individual with cutaneous T-cell lymphoma (CTCL).
    Williams K; Gibson A; McNamara L; Jones T; Lloyd AJ
    J Med Econ; 2020 Oct; 23(10):1142-1150. PubMed ID: 32644862
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom.
    Hubig L; Sussex AK; MacCulloch A; Hughes D; Graham R; Morris L; Raza S; Lloyd AJ; Sowinski A; Gallop K
    J Health Econ Outcomes Res; 2023; 10(1):41-50. PubMed ID: 36883056
    [No Abstract]   [Full Text] [Related]  

  • 7. Diagnosing X-linked Myotubular Myopathy - A German 20-year Follow Up Experience.
    Gangfuss A; Schmitt D; Roos A; Braun F; Annoussamy M; Servais L; Schara-Schmidt U
    J Neuromuscul Dis; 2021; 8(1):79-90. PubMed ID: 33164942
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
    Duong T; Harding G; Mannix S; Abel C; Phillips D; Alfano LN; Bönnemann CG; Lilien C; Lowes LP; Servais L; Warken-Madelung B; Nieto Bergman S; James ES; Noursalehi M; Prasad S; Rico S; Bilder DA
    J Neuromuscul Dis; 2021; 8(1):63-77. PubMed ID: 32925083
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Real-world analysis of healthcare resource utilization by patients with X-linked myotubular myopathy (XLMTM) in the United States.
    Graham RJ; Darras BT; Haselkorn T; Fisher D; Genetti CA; Miller W; Beggs AH
    Orphanet J Rare Dis; 2023 Jun; 18(1):138. PubMed ID: 37280644
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Gait characteristics in a canine model of X-linked myotubular myopathy.
    Goddard MA; Burlingame E; Beggs AH; Buj-Bello A; Childers MK; Marsh AP; Kelly VE
    J Neurol Sci; 2014 Nov; 346(1-2):221-6. PubMed ID: 25281397
    [TBL] [Abstract][Full Text] [Related]  

  • 11. X-linked myotubular myopathy: A prospective international natural history study.
    Annoussamy M; Lilien C; Gidaro T; Gargaun E; Chê V; Schara U; Gangfuß A; D'Amico A; Dowling JJ; Darras BT; Daron A; Hernandez A; de Lattre C; Arnal JM; Mayer M; Cuisset JM; Vuillerot C; Fontaine S; Bellance R; Biancalana V; Buj-Bello A; Hogrel JY; Landy H; Servais L
    Neurology; 2019 Apr; 92(16):e1852-e1867. PubMed ID: 30902907
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Successful use of non-invasive positive pressure ventilation in a patient with the severe form of X-linked myotubular myopathy.
    Inoue K; Kumada T; Hiejima I; Fujii T
    Brain Dev; 2018 May; 40(5):421-424. PubMed ID: 29343419
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis.
    Graham RJ; Muntoni F; Hughes I; Yum SW; Kuntz NL; Yang ML; Byrne BJ; Prasad S; Alvarez R; Genetti CA; Haselkorn T; James ES; LaRusso LB; Noursalehi M; Rico S; Beggs AH
    Arch Dis Child; 2020 Apr; 105(4):332-338. PubMed ID: 31484632
    [TBL] [Abstract][Full Text] [Related]  

  • 14. rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy.
    Ross JA; Tasfaout H; Levy Y; Morgan J; Cowling BS; Laporte J; Zanoteli E; Romero NB; Lowe DA; Jungbluth H; Lawlor MW; Mack DL; Ochala J
    Acta Neuropathol Commun; 2020 Oct; 8(1):167. PubMed ID: 33076971
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Cognitive, adaptive and perseverative aspects characterization of children with XLMTM: An explorative study.
    Cumbo F; Tosi M; Mizzoni I; Catteruccia M; Carlesi A; Bertini E; D'Amico A
    Eur J Paediatr Neurol; 2024 Jul; 51():58-61. PubMed ID: 38824722
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Estimation of utilities in attention-deficit hyperactivity disorder for economic evaluations.
    Lloyd A; Hodgkins P; Sasane R; Akehurst R; Sonuga-Barke EJ; Fitzgerald P; Nixon A; Erder H; Brazier J
    Patient; 2011; 4(4):247-57. PubMed ID: 21995830
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Affected female carriers of MTM1 mutations display a wide spectrum of clinical and pathological involvement: delineating diagnostic clues.
    Biancalana V; Scheidecker S; Miguet M; Laquerrière A; Romero NB; Stojkovic T; Abath Neto O; Mercier S; Voermans N; Tanner L; Rogers C; Ollagnon-Roman E; Roper H; Boutte C; Ben-Shachar S; Lornage X; Vasli N; Schaefer E; Laforet P; Pouget J; Moerman A; Pasquier L; Marcorelle P; Magot A; Küsters B; Streichenberger N; Tranchant C; Dondaine N; Schneider R; Gasnier C; Calmels N; Kremer V; Nguyen K; Perrier J; Kamsteeg EJ; Carlier P; Carlier RY; Thompson J; Boland A; Deleuze JF; Fardeau M; Zanoteli E; Eymard B; Laporte J
    Acta Neuropathol; 2017 Dec; 134(6):889-904. PubMed ID: 28685322
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report.
    Molera C; Sarishvili T; Nascimento A; Rtskhiladze I; Muñoz Bartolo G; Fernández Cebrián S; Valverde Fernández J; Muñoz Cabello B; Graham RJ; Miller W; Sepulveda B; Kamath BM; Meng H; Lawlor MW
    J Neuromuscul Dis; 2022; 9(1):73-82. PubMed ID: 34366366
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Parallel Valuation of the EQ-5D-3L and EQ-5D-5L by Time Trade-Off in Hungary.
    Rencz F; Brodszky V; Gulácsi L; Golicki D; Ruzsa G; Pickard AS; Law EH; Péntek M
    Value Health; 2020 Sep; 23(9):1235-1245. PubMed ID: 32940242
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.
    Sarwal A; Cartwright MS; Walker FO; Mitchell E; Buj-Bello A; Beggs AH; Childers MK
    Muscle Nerve; 2014 Oct; 50(4):607-9. PubMed ID: 24861988
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 7.