Results 131 to 140 of about 169,190,458 (279)
IDB Newsletter: Vol. 8, no. 4, October 1970
, 1970 IDB Newsletter is a monthly publication of the Inter-American Development that reports on the activities of the Inter-American Development Bank and appears in English and Spanish ...
Inter-American Development Bank
core +1 more source
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw +5 more
wiley +1 more source
IDB Newsletter: Vol. 6, no. 1, January 1968
, 1968 IDB Newsletter is a monthly publication of the Inter-American Development that reports on the activities of the Inter-American Development Bank and appears in English and Spanish ...
Inter-American Development Bank
core +1 more source
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit +11 more
wiley +1 more source
IDB Newsletter: Vol. 6, no. 3, July 1968
, 1968 IDB Newsletter is a monthly publication of the Inter-American Development that reports on the activities of the Inter-American Development Bank and appears in English and Spanish ...
Inter-American Development Bank
core +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
IDB Newsletter: Vol. 8, no. 3, July 1970
, 1970 IDB Newsletter is a monthly publication of the Inter-American Development that reports on the activities of the Inter-American Development Bank and appears in English and Spanish ...
Inter-American Development Bank
core +1 more source
A Two‐Stage Questionnaire and Actigraphy Screening for iRBD in a Multicenter Retrospective Cohort
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Isolated rapid‐eye‐movement sleep behavior disorder is a prodromal marker of synucleinopathies. However, most cases remain undiagnosed due to the insufficient predictive value of questionnaires and limited access to confirmatory video‐polysomnography. We assessed a two‐stage screening strategy combining a brief questionnaire on rapid‐
Caleb A. Massimi +17 more
wiley +1 more source
Challenging Inequalities: Our Early Childhood Development Sector Framework Document at a Glance
Investing in the first five years of life is essential for early childhood development (ECD) and contributes to increasing societal productivity, reducing poverty, and promoting equality.
Inter-American Development Bank
core +1 more source
IDB Newsletter: Vol. 7, no. 2, April 1969
, 1969 IDB Newsletter is a monthly publication of the Inter-American Development that reports on the activities of the Inter-American Development Bank and appears in English and Spanish ...
Inter-American Development Bank
core +1 more source