Neurofibromatosis
FDA approves Gomekli for neurofibromatosis type 1 with plexiform neurofibromas
The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.
Facial involvement of cutaneous neurofibromas represent largest quality of life impact
Patients with increasing numbers of cutaneous neurofibromas experienced a growing negative impact on their quality of life, especially if there was facial involvement, according to a study.
FDA grants Ojemda accelerated approval for children with advanced low-grade brain cancer
The FDA granted accelerated approval to Ojemda for the treatment of certain children with relapsed or refractory low-grade glioma.
Nonsurgical treatments may be effective option for cutaneous neurofibromas
Minimally invasive, nonsurgical local treatments could be a possible option for small cutaneous neurofibromas instead of surgical removal, according to a study.
Mirdametinib improves neurofibromatosis type 1-associated plexiform neurofibromas
Positive results from a pivotal phase 2b trial showed mirdametinib significantly improved the outcomes of patients with neurofibromatosis type 1-associated plexiform neurofibromas, SpringWorks Therapeutics announced in a press release.
Patients who visited neurofibromatosis specialists received better guided care
PHILADELPHIA — Patients who availed themselves of specialized care for neurofibromatosis received more guideline-concordant care than other patients, with differences found along racial and economic lines, per a poster presentation.
Neurofibromatosis type 1 tied to severe auditory dysfunction in children, adults
Severe auditory dysfunction was a common neurobiological feature of neurofibromatosis type 1, according to results of a case-control study published in JAMA Network Open.