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Diffuse Intrinsic Pontine Glioma

Phase I Study of Autologous T Lymphocytes Expressing GD2-specific Chimeric Antigen and Constitutively Active IL-7 Receptors for the Treatment of Patients With GD2-expressing Brain Tumors (GAIL-B) C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B) Phase 1 DESCRIPTION: Researchers in Texas Children's Hospital are seeking for patients with high grade glioma (HGG)...

Legislation to Help Diagnose Children with Rare Diseases Introduced

Washington, D.C.—U.S. Senators Susan Collins (R-ME), Doug Jones (D-AL), Martha McSally (R-AZ), and Bob Menendez (D-NJ) introduced bipartisan legislation to help thousands of children with rare diseases receive a diagnosis and proper treatment more quickly. Children with rare diseases face a “diagnostic odyssey” that typically lasts...

Novartis plans to give away world’s costliest therapy to some patients

Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug program that one patient group worried was a “health lottery” that could neglect some babies. Starting Jan. 2, Novartis’s AveXis unit which developed Zolgensma...