Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy.

Context: Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial.

Four Cases of Myeloproliferative Disorders Associated With Down Syndrome: Distinguishing ML-DS From TAM-DS: Distinguishing TAM-DS and ML-DS: Report of 4 Cases.

Down syndrome (DS) is defined by an extra copy of chromosome 21 and confers an increased susceptibility to hematological disorders. Transient abnormal myelopoiesis (TAM) and myeloid-leukemia associated with Down syndrome (ML-DS) are two conditions that need to be accurately diagnosed to provide appropriate management. Both TAM and ML-DS are characterized by proliferation of megakaryoblasts carrying a mutation in the GATA1 gene.

Experience and andrological follow-up after testicular tissue cryopreservation.

Research Question: What is the experience and mid- and long-term andrological health follow-up of (pre)pubertal males who have undergone testicular tissue freezing (TTF)?

Design: This single-centre longitudinal retrospective cohort study reports on the mid- and long-term andrological health follow-up of (pre)pubertal males and young adults who underwent TTF for fertility preservation between January 2007 and December 2018. Medical characteristics and questionnaire data collected more than 18 months after TTF were analysed.

Results: Thirty-six patients were revisited during a medical follow-up consultation.

Chemo-, Regio-, and Stereoselective -Hydroboration of 1,3-Enynes: Copper-Catalyzed Access to ()- and ()-2-Boryl-1,3-dienes.

A copper-catalyzed alkyne-selective hydroboration of 1,3-enynes is disclosed, providing access to the previously elusive 2-boryl-1,3-dienes. Using CuOAc, Xantphos, and HBpin, Bpin was installed on the internal carbon of a series of symmetric and nonsymmetric 1,3-enynes, affording products with excellent : selectivity. The utility of the 2-boryl-1,3-diene products was demonstrated by transformation to useful functional groups.