case study

Patient Reports

There are a lot of impressive stories about how “the rare ones” deal with their daily challenges

Situation

This survey, conducted between January and March 2018 in 13 European countries, sought to evaluate the awareness and access to mexiletine and subsequent harm caused by limited treatment access.

Our Solution

  • Qualitative semi-structured interviews with patient organisations or independent patient representatives and myotonia specialists. The interviews, using a bespoke questionnaire (10) , were focused on obtaining data on the overall treatment of NDM, and the outcomes for patients not having access to treatment.
  • Quantitative online-survey (11) that was aimed at verifying the data collected in the interviews and further establishing the diagnosis, symptoms, treatments and access to treatments for patients with NDM.

Results

The online patient survey included 37 participants with confirmed MC in 6 European countries. Of the patients taking mexiletine, 84% had been prescribed mexiletine by a neurologist. Other patients were prescribed mexiletine by cardiologists, general practitioners, ophthalmologists, pneumologist and rheumatologists. Difficulty in obtaining mexiletine was experienced (multiple answers applied to some patients)