Cerliponase alfa is the first FDA-approved treatment to slow loss of walking ability (ambulation) in symptomatic pediatric patients three years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.The efficacy of cerliponase alfa was established in a non-randomized, single-arm dose escalation clinical study in 22 symptomatic pediatric patients with CLN2 disease and compared to 42 untreated patients with CLN2 disease from a natural history cohort (an independent historical control group) who were at least three years old and had motor or language symptoms. Taking into account age, baseline walking ability and genotype, cerliponase alfa-treated patients demonstrated fewer declines in walking ability compared to untreated patients in the natural history cohort.The safety of cerliponase alfa was evaluated in 24 patients with CLN2 disease aged three to eight years who received at least one dose of cerliponase alfa in clinical studies. The trial was conducted in the United States, United Kingdom, Germany and Italy. The safety and effectiveness of cerliponase alfa has not been established in patients less than three years of age.Brineura-treated patients were compared to untreated patients from a natural history cohort by assessing disease progression through Week 96 of treatment. The investigators measured the loss of ability to walk or crawl using the Motor domain of the CLN2 Clinical Rating Scale.
Granuloma gluteale infantum is a cutaneous condition that appears in the anogenital region of infants as a complication of diaper dermatitis.According to some, no granulomas are found. See also
Superficial granulomatous pyoderma
List of cutaneous conditions

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