CNW/ - Alexion, AstraZeneca Rare Disease's Koselugo (selumetinib), an oral, selective MEK inhibitor, has been approved in ...

As a new year begins, plans are underway for the 8 th Annual Rare Disease Genomics Symposium to be held on February 26 th and 27 th. The two-day event will be virtual this year to limit the spread of ...

A Media Snippet accompanying this announcement is available by clicking on this link.

Almost half of adults and children with neurofibromatosis type 1 (NF1) responded to the investigational MEK inhibitor mirdametinib, a large multicenter study of the rare condition showed. Overall, 53 ...

GOMEKLI is the first FDA-approved treatment for adult and pediatric NF1-PN, showing effective tumor reduction and manageable safety. SpringWorks Therapeutics has announced the FDA approval of GOMEKLI ...

Gomekli (mirdametinib) is the first treatment that is FDA approved for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN). Selumetinib (Koselugo), a ...

Cred: SpringWorks Therapeutics. Mirdametinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2. Gomekli is the first medication approved for both adult and pediatric patients with ...

– If approved, mirdametinib is expected to be the first and only therapy in the European Union with marketing authorization for both adults and children with NF1-PN – – Decision from European ...

Q: A doctor said the rashes on my son's back were “coffee spots” and that meant he might have neurofibromatosis. I read there is a new treatment for this condition; is it a cure? A: Café-au-lait spots ...