The FDA approved Daybue on March 10, 2023, for the treatment of Rett syndrome in adults and children aged 2 years and older.

The FDA has approved BioMarin's BRINEURA® (cerliponase alfa) for treating children under 3 years old with CLN2 disease.

The FDA approved Skyclarys on February 28, 2023, for the treatment of Friedreich's ataxia in adults and teenagers 16 years and older.

Jul 31st latest updates in the global new drug development field, including progress in new drug research and development, transaction information, and partnership developments.

The FDA approved Prevduo for use in adults and children at least 2 years old to help regain muscle function after surgery.

IASO Bio Receives NMPA Clearance for IASO-782 IND Application to Address Systemic Lupus Erythematosus (SLE).

The FDA granted approval to Altuviiio as a first-in-class, high-sustained factor VIII replacement therapy for hemophilia A patients on February 22, 2023.

Nkarta Reveals Start of Clinical Trial Led by Investigators for NKX019 in Treating Systemic Lupus Erythematosus.

The FDA granted accelerated approval to Sparsentan for its ability to reduce proteinuria (excess protein in the urine) in adults with primary immunoglobulin A nephropathy.

Inimmune reports initial cancer patient treated in a phase 1 trial with its new TLR7/8 agonist immunotherapy, INI-4001.

Pirtobrutinib, marketed under the brand name Jaypirca, is FDA approved. The FDA granted accelerated approval to Pirtobrutinib on January 27, 2023.

Transposon Reports Conclusive Data from Phase 2 Trial of TPN-101 Targeting C9orf72-Linked ALS and/or Frontotemporal Dementia.