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Intrathecal Gene Therapy Shows Promise in Giant Axonal Neuropathy

by HealthDay Reporter | Apr 22, 2024

Intrathecal gene transfer with scAAV9/JeT-GAN may show some benefit in motor function for children with giant axonal neuropathy.     Intrathecal gene transfer with scAAV9/JeT-GAN may result in some benefit for children with giant axonal neuropathy, according...

Survival 21 Percent a Year After ICU Discharge for Blood Cancer Patients

by HealthDay Reporter | Apr 18, 2024

Acute respiratory failure and sepsis were the most common reasons for ICU admission.     For hematologic malignancy (HM) patients admitted to the intensive care unit (ICU), survival is 49 percent at seven days and 21 percent at 12 months, according to a...

CARv3-TEAM-E T-Cell Treatment Beneficial for Recurrent Glioblastoma

by HealthDay Reporter | Apr 16, 2024

Dramatic and rapid radiographic tumor regression seen in three cases of recurrent glioblastoma; transient responses seen in two patients.     For patients with recurrent glioblastoma, treatment with chimeric antigen receptor (CAR) T-cells engineered to...

Second Primary Malignancies Represent Small Proportion of Adverse Events After CAR T-Cell Therapy

by HealthDay Reporter | Apr 15, 2024

Most reports of SPMs included axicabtagene ciloleucel and tisagenlecleucel; most frequent SPMs were leukemias.     Second primary malignancies (SPMs) represent a small proportion of adverse events (AEs) reported by patients receiving chimeric antigen...

Many Children With Sickle Cell Anemia Do Not Meet Quality Indicators

by HealthDay Reporter | Apr 11, 2024

Rates of receipt of recommended antibiotic prophylaxis and annual transcranial Doppler low.     A number of children with sickle cell anemia (SCA) have low rates of receipt of recommended antibiotic prophylaxis and annual transcranial Doppler (TCD)...
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