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Survey on transition from paediatric to adult care among young people living with Duchenne

August 23rd, 2022

 

 

 

 

 

 

 

 

 

Have your say on moving into adulthood. We wish to share a survey from PTC Therapeutics to get answers to important questions about transition from paediatric to adult care among young people living with Duchenne to better understand the transition experience between paediatric and adult services for DMD.

PTC would like to find out opinions from the following people:

1) Teenagers and young people between 16 and 26 years of age living with Duchenne who:

  • are about to begin transition between paediatric and adult care
  • have recently transitioned between paediatric and adult care.

2) Parent and carers of the teenagers and young people living with Duchenne.

As a thank you for your time you will receive a €6 Amazon gift card.

Take the survey here.

 

About filling in the survey

In section 1, please select one of these options:

  1. Pre-transition
  2. Pre-transition parent/carer
  3. Post-transition
  4. Post-transition parent/carer

If you select option ‘e. Other’ the survey will end and you will not be able to continue.

Thank you so much for taking the time to your help shape transition for the future!

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Categories: Research

1. Muscular Dystrophies

  • Becker muscular dystrophy
  • Duchenne muscular dystrophy
  • Manifesting carrier of Duchenne
  • Congenital muscular dystrophy
  •     •  General
  •     •  MDC1A (merosin-deficient congenital muscular dystrophy)
  •     •  Rigid spine syndrome (RSS)
  •     •  Ullrich congenital muscular dystrophies
  •     •  Bethlem myopathy
  • Emery-Dreifuss muscular dystrophy
  • Facioscapulohumeral muscular dystrophy
  • Limb-girdle types of muscular dystrophy (LGMD)
  •     •  General
  •     •  LGMD 1B (also known as Laminopathy)
  •     •  LGMD 1C (also known as Caveolinopathy)
  •     •  LGMD 2A (also known as Calpainopathy)
  •     •  LGMD 2B (also known as Dysferlinopathy)
  •     •  LGMD 2I
  • Ocular myopathies including ocularopharangeal muscular dystrophy

2. Myotonic Disorders

  • Congenital Myotonic Dystrophy
  • Myotonia
  • Myotonic Dystrophy

3. Congenital Myopathies

  • Central Core Myopathy
  • Congenital Fibre-type Disproportion Myopathy
  • Minicore (Multicore) myopathy
  • Myotubular or Centronuclear myopathy
  • Nemaline myopathy

4. Mitochondrial Myopathies

  • Mitochondrial Myopathies

5. Metabolic Disorders

  • Metabolic disorders (general)
  • McArdle’s Disease
  • Pompe’s Disease

6. Periodic Paralyses

  • Periodic Paralyses

7. Autoimmune Myositis

  • Polymyositis, Dermatomyositis and Sarcoid myopathy
  • Juvenile dermatomyositis
  • Inclusion body myositis

8. Spinal Muscular Atrophies

  • Severe (Type I)
  • Intermediate (Type II)
  • Mild (Type III)
  • Adult spinal muscular atrophy

9. Hereditary Motor and Sensory Neuropathies

  • (Also known as Charcot-Marie-Tooth or Peroneal muscular atrophy)

10. Disorders of the Neuromuscular Junction

  • Congenital myasthenic syndromes
  • Myasthenia Gravis

11. Friedreich’s Ataxia

  • Friedreich’s Ataxia

12. Other (Please Specify)

13. Unspecified