For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Dr. Matthew Matasar discusses why stem cell transplant is still a standard, life-saving option in blood cancers and how CAR-T ...

Kids with a rare bone cancer called Ewing sarcoma might live longer if prescribed pazopanib, a drug that blocks blood supply ...

AGC Biologics, your friendly CDMO expert, announced an agreement to provide comprehensive development and manufacturing for Rarity PBC's gene therapy.

The FDA has accepted Pharming Group’s supplemental new drug application for leniolisib in treating activated phosphoinositide ...

Key Takeaways An already approved drug might help kids with a rare bone cancerPazopanib extended survival in a small group of ...

A Welsh boy with a rare and life-threatening condition "danced his way through" treatment on the "bubble ward" at the Royal Victoria Infirmary. Little Jasper Hodgson Smith, now 8, is one of just 35 ...

Twenty-month-old Vida Rodriguez is the reason her 7-year-old sister, Alessia, is playing at home — instead of lying in a ...

Survival rates from small trial suggest that adding pazopanib to standard treatment for Ewing sarcoma in children may extend lives without adding severe toxicity.

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

Ewing sarcoma is one of the most common bone cancers seen in children, and if it spreads, it can be deadly. One study found ...

Like the surface of the moon, new research published today in Cell finds the existence of craters on the surface of melanoma ...