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Motor neurone disease: progress and challenges.

Abstract Major progress has been made over the past decade in the understanding of motor neurone disease (MND), changing the landscape of this complex disease. Through identifying positive prognostic factors, new evidence-based standards of care have been established that improve patient survival, reduce burden of disease for patients and their carers, and enhance quality of life. These factors include early management of respiratory dysfunction with non-invasive ventilation, maintenance of weight and nutritional status, as well as instigation of a multidisciplinary team including neurologists, general practitioners and allied health professionals. Advances in technology have enhanced our understanding of the genetic architecture of MND considerably, with implications for patients, their families and clinicians. Recognition of extra-motor involvement, particularly cognitive dysfunction, has identified a spectrum of disease from MND through to frontotemporal dementia. Although riluzole remains the only disease-modifying medication available in clinical practice in Australia, several new therapies are undergoing clinical trials nationally and globally, representing a shift in treatment paradigms. Successful translation of this clinical research through growth in community funding, awareness and national MND research organisations has laid the foundation for closing the research-practice gap on this debilitating disease. In this review, we highlight these recent developments, which have transformed treatment, augmented novel therapeutic platforms, and established a nexus between research and the MND community. This era of change is of significant relevance to both specialists and general practitioners who remain integral to the care of patients with MND.
PMID
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Authors

Mayor MeshTerms

Interdisciplinary Communication

Standard of Care

Keywords
Journal Title the medical journal of australia
Publication Year Start




PMID- 28446118
OWN - NLM
STAT- MEDLINE
DA  - 20170427
DCOM- 20170502
LR  - 20170502
IS  - 1326-5377 (Electronic)
IS  - 0025-729X (Linking)
VI  - 206
IP  - 8
DP  - 2017 May 01
TI  - Motor neurone disease: progress and challenges.
PG  - 357-362
AB  - Major progress has been made over the past decade in the understanding of motor
      neurone disease (MND), changing the landscape of this complex disease. Through
      identifying positive prognostic factors, new evidence-based standards of care
      have been established that improve patient survival, reduce burden of disease for
      patients and their carers, and enhance quality of life. These factors include
      early management of respiratory dysfunction with non-invasive ventilation,
      maintenance of weight and nutritional status, as well as instigation of a
      multidisciplinary team including neurologists, general practitioners and allied
      health professionals. Advances in technology have enhanced our understanding of
      the genetic architecture of MND considerably, with implications for patients,
      their families and clinicians. Recognition of extra-motor involvement,
      particularly cognitive dysfunction, has identified a spectrum of disease from MND
      through to frontotemporal dementia. Although riluzole remains the only
      disease-modifying medication available in clinical practice in Australia, several
      new therapies are undergoing clinical trials nationally and globally,
      representing a shift in treatment paradigms. Successful translation of this
      clinical research through growth in community funding, awareness and national MND
      research organisations has laid the foundation for closing the research-practice 
      gap on this debilitating disease. In this review, we highlight these recent
      developments, which have transformed treatment, augmented novel therapeutic
      platforms, and established a nexus between research and the MND community. This
      era of change is of significant relevance to both specialists and general
      practitioners who remain integral to the care of patients with MND.
FAU - Dharmadasa, Thanuja
AU  - Dharmadasa T
AD  - Brain and Mind Centre, University of Sydney, Sydney, NSW
      [email protected]
FAU - Henderson, Robert D
AU  - Henderson RD
AD  - Royal Brisbane and Women's Hospital, Brisbane, QLD.
FAU - Talman, Paul S
AU  - Talman PS
AD  - Geelong Hospital, Geelong, VIC.
FAU - Macdonell, Richard Al
AU  - Macdonell RA
AD  - University of Melbourne, Melbourne, VIC.
FAU - Mathers, Susan
AU  - Mathers S
AD  - Calvary Health Care Bethlehem, Melbourne, VIC.
FAU - Schultz, David W
AU  - Schultz DW
AD  - Flinders Medical Centre, Adelaide, SA.
FAU - Needham, Merrillee
AU  - Needham M
AD  - Western Australian Neuromuscular Research Institute, Perth, WA.
FAU - Zoing, Margaret
AU  - Zoing M
AD  - Brain and Mind Centre, University of Sydney, Sydney, NSW.
FAU - Vucic, Steve
AU  - Vucic S
AD  - Westmead Hospital, Sydney, NSW.
FAU - Kiernan, Matthew C
AU  - Kiernan MC
AD  - Brain and Mind Centre, University of Sydney, Sydney, NSW.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - Australia
TA  - Med J Aust
JT  - The Medical journal of Australia
JID - 0400714
SB  - IM
MH  - Australia
MH  - Evidence-Based Practice
MH  - Genetic Testing
MH  - Genetic Therapy
MH  - Humans
MH  - *Interdisciplinary Communication
MH  - Motor Neuron Disease/*therapy
MH  - Neuroprotection
MH  - Noninvasive Ventilation
MH  - Nutritional Support
MH  - Patient Acceptance of Health Care
MH  - Quality of Life
MH  - Randomized Controlled Trials as Topic
MH  - *Standard of Care
MH  - Stem Cell Transplantation
EDAT- 2017/04/28 06:00
MHDA- 2017/05/04 06:00
CRDT- 2017/04/28 06:00
PHST- 2016/09/06 [received]
PHST- 2017/01/19 [accepted]
AID - 10.5694/mja16.01063 [pii]
PST - ppublish
SO  - Med J Aust. 2017 May 1;206(8):357-362.

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