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Prolonged prevention of autoimmune diabetes
Teplizumab was previously shown in a clinical trial to delay onset of type 1 diabetes (T1D) in high-risk relatives of individuals with T1D. Now, Sims
et al. extend the follow-up analysis of this trial by 12 months, finding that efficacy of the initial 2-week treatment course persisted, with an extended time to T1D diagnosis in the teplizumab-treated group. Clinical benefits associated with reversed C-peptide decline improved beta cell function and partial exhaustion in CD8
+ T cells in the treated patients. HbA1c did not differ between placebo and treatment groups. This follow-up study further supports the use of anti-CD3 treatment for the prevention of T1D.
Teplizumab improves and stabilizes beta cell function in antibody-positive high-risk individuals
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NIH effort seeks to understand MIS-C, range of SARS-CoV-2 effects on children
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02 marzo 2021 17:14
Fonte: Adnkronos
#salute-e-benessere
AUSTIN, Texas, March 2, 2021 /PRNewswire/ Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.
CLN3, one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), is a rare, fatal, inherited, neurodegenerative disease for which there is no treatment. Since it was founded in 2008, BBDF has spearheaded the development of BBDF-101.