Evaluations of novel approaches to treating NF-1 tumors are underwayOctober 29, 2021Rare DiseasesDermatology
Clinical genetic testing for skin disorders continues to advanceSeptember 8, 2021Rare DiseasesDermatology
Targeted therapies for vascular anomalies continue to be refinedSeptember 3, 2021DermatologyRare Diseases
FDA approves first once-weekly growth hormone for childrenSeptember 3, 2021EndocrinologyRare Diseases
Study informs about risks of discontinuing meds in JIAAugust 25, 2021Rare DiseasesMusculoskeletal Disorders
Novel gene therapy ‘reprograms’ cells to reverse neurologic deficits in children with rare diseaseJuly 22, 2021Rare DiseasesNeurology
U.S., international MIS-C studies yield disparate resultsJune 18, 2021COVID-19 UpdatesInfectious DiseasesRare DiseasesCardiologyPulmonologyImmunology
Secukinumab provides clinical benefit in phase 3 juvenile arthritis trialJune 15, 2021Rare DiseasesMusculoskeletal Disorders
EULAR, ACR present preliminary recommendations for rare genetic autoinflammatory diseasesJune 5, 2021Rare Diseases
Sickle cell disease: Epidemiological change in bacterial infectionsJune 1, 2021Infectious DiseasesRare Diseases
Two treatments show early promise for hypothalamic obesityMay 10, 2021ObesityEndocrinologyRare DiseasesMixed Topics
