Doctor in the House: A New Treatment for Neurofibromatosis in Malaysia

In April, the Ministry of Health announced that it has given the “orphan medicine” status to Koselugo, a prescription drug used in treating children with neurofibromatosis type 1 and the only medical treatment that is available for this rare disorder. With May being Neurofibromatosis Awareness Month, we discuss what the availability of this treatment means for children with neurofibromatosis and the gaps that still remain, together with Prof Dr Thong Meow Keong, consultant paediatrician & clinical geneticist, and Carol Lim, who is living with neurofibromatosis.