Therapeutic targets
- Relief of symptoms
- Attempt to delay the progression of the disease
Therapy recommendations
- A causal therapy does not exist to date.
- The following drugs are being used in trials:
- Benzodiazepines such as clonazepam or antiepileptic drugs such as valproate for myclonias (muscle twitching); good response, especially in the initial phase of disease
- Flupirtine* (centrally acting, non-opioid analgesic) – to slow cognitive decline.
- Doxycycline (tetracycline) – reported to double survival (?) [observational studies].
* Pharmacovigilance Risk Assessment Committee (PRAC) recommends withdrawal of marketing approval (2018) due to severe liver damage; approval of flupirtine-containing drugs revoked EU-wide (2018).
Note: Red-hand letter (AkdÄ Drug Safety Mail): new contraindications, strengthened warnings, and measures to avoid exposure to valproate during pregnancy:
- In girls and women of childbearing age, valproate may be used only if other treatments are not effective or are not tolerated.
- Valproate is contraindicated in women of childbearing age unless the pregnancy prevention program is followed.
- Valproate is contraindicated in epilepsy during pregnancy unless no suitable alternatives are available.
- Valproate is contraindicated during pregnancy for bipolar disorder and migraine prophylaxis.
