Orphanet: Search for an orphan drug
x

Search for an orphan drug

* (*) mandatory field

Other search option(s)

Marketing authorisation with orphan designation - Europe

Therapeutic indication

As monotherapy for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.

Diseases list

The documents contained in this web site are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment.