A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy.

Objective: To investigate the efficacy and safety of aceneuramic acid extended-release (Ace-ER), a treatment intended to replace deficient sialic acid, in patients with GNE myopathy.

Methods: UX001-CL301 was a phase 3, double-blind, placebo-controlled, randomized, international study evaluating the efficacy and safety of Ace-ER in patients with GNE myopathy. Participants who could walk ≥200 meters in a 6-minute walk test at screening were randomized 1:1, and stratified by sex, to receive Ace-ER 6 g/d or placebo for 48 weeks and assessed every 8 weeks.

Safety of MR Imaging at 1.5 T in Fetuses: A Retrospective Case-Control Study of Birth Weights and the Effects of Acoustic Noise.

Purpose: To evaluate the effects of exposure to routine magnetic resonance (MR) imaging at 1.5 T during pregnancy on fetal growth and neonatal hearing function in relation to the dose and timing of in utero exposure in a group of newborns at low risk for congenital hearing impairment or deafness.

Materials And Methods: This retrospective case-control study was approved by the local ethics committee, and written informed consent was waived.

[Treatment of cleft lip and palate in the children's Hospital Reine Fabiola in Bruxelles].

The priority in the treatment of facial clefts is to avoid the iatrogenic sequellae by restoring a normal anatomy of the face. The difficulty of this treatment is that the majority of the sequellae are only observed 15 years after the primary treatment. After having treated our children during more than 20 years by a primary closure of the lip at 6 months and closure of the palate by push back according to Veau Wardill at 18 months, we adopted the technique of René Malek since 1981 with early closure of the palate at 3 months then, without undermining of the palatal mucosa, closure of the bony palate with a vomer flap at the age of 6 months.