Category Archives: Congenital disorders
Congenital disorders
Born with a heart malformation: The children forgotten by the Mexican health system
Oct
When Nevith was five years old, her heart began to hurt. She used to tell her mother that “it beat fast, as if it was going to jump out of her little chest. When she walked fast her heartbeat quickened, but sometimes it also happened when she was asleep, and she woke up scared,” says […]
PHOENIX, Ariz., Oct. 26, 2023 /PRNewswire/ — Phoenix Children’s is taking another leap forward with the addition of two internationally recognized scientists and new research efforts that will improve quality and outcomes for children facing a range of illnesses. “We are thrilled to welcome two brilliant researchers whose knowledge and expertise expand our purview and add […]
Congenital disorders
Rivogenlecleucel by Bellicum Pharmaceuticals for Osteopetrosis: Likelihood of Approval
Oct
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Rivogenlecleucel overview Rivogenlecleucel (BPX-501) is under development for the treatment of […]
Congenital disorders
Rivogenlecleucel by Bellicum Pharmaceuticals for Aplastic Anemia: Likelihood of Approval
Oct
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Rivogenlecleucel overview Rivogenlecleucel (BPX-501) is under development for the treatment of […]
Congenital disorders
Trehalose by Seelos Therapeutics for Oculopharyngeal Muscular Dystrophy: Likelihood of Approval
Oct
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. Trehalose overview Trehalose is under development for the treatment of oculopharyngeal […]
Congenital disorders
Deaths due to homicide are the second-highest cause of injury-related mortality in children aged one to 14, audit finds
Oct
Deaths due to homicide or filicide accounted for 17.8pc of all injury-related deaths during 2019 and 2021, compared with 6.6pc between 2007 and 2018. The vast majority of these deaths occurred in the child’s own home and were committed by a family member or someone known to the child. The National Paediatric Mortality Register (NPMR) […]
Congenital disorders
Bi-allelic truncating variants in CASP2 underlie a neurodevelopmental disorder with lissencephaly
Oct
Abstract Lissencephaly (LIS) is a malformation of cortical development due to deficient neuronal migration and abnormal formation of cerebral convolutions or gyri. Thirty-one LIS-associated genes have been previously described. Recently, biallelic pathogenic variants in CRADD and PIDD1, have associated with LIS impacting the previously established role of the PIDDosome in activating caspase-2. In this report, […]