January 09, 2026

Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura

The FDA's approval of recombinant ADAMTS13 marks a significant advancement in managing congenital thrombotic thrombocytopenic purpura, ensuring better care for patients suffering from this rare condition.

JADPRO

January 08, 2026

Non–Total Body Irradiation Conditioning Regimen for Young Patients With MRD-Negative B-ALL

Using a chemotherapy-based conditioning regimen led to 2-year event-free survival rates comparable to that of total body irradiation–based conditioning regimens in pediatric and young adult patients w...

The ASCO Post

December 25, 2025

Lasting Retinal Effects in Preterm Babies

The long shadow of prematurity: what adult retinas reveal decades later

The Ophthalmologist

December 23, 2025

Rare Disease Reanalysis Ended Our 5-Year Wait

In conversation with the clinician who developed the proactive exome reanalysis approach, and the mother whose child benefited from it

The Pathologist

December 22, 2025

Eliminating Barriers to Care for Patients With Intellectual and/or Developmental Disabilities

A recent large, retrospective study by Giblon et al has found that individuals with intellectual and/or developmental disabilities face an increased risk of developing cancer, higher symptom burden on...

The ASCO Post

December 17, 2025

Promising Data For Off-the-Shelf Gene-edited Cell Therapy

New clinical results outline patient responses to a donor-derived, gene-edited cell therapy in relapsed and refractory T-cell leukemia

The Medicine Maker

December 12, 2025

Acupuncture May Improve Perceived Cognitive Difficulties in Breast Cancer Survivors

New findings from the phase II ENHANCE clinical trial indicate that both real and sham acupuncture improve breast cancer survivors’ perceived cognitive impairment more effectively than usual care alon...

The ASCO Post

December 11, 2025

Myopia: A Public Health Emergency

Global experts declare childhood myopia a public health emergency in HOYA-led call to action

The Ophthalmologist

December 10, 2025

This Week’s CGT News: Vertex’s Gene Therapies Show Promise in Younger Children

Our latest news roundup reports on new data for Vertex’s exa-cel, funding for a cell therapy for breast cancer, and new technologies for manufacturing

The Medicine Maker