European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...

ImCheck’s ICT01 Receives FDA Orphan Drug Designation for Treatment of Acute Myeloid Leukemia Clinical data showing ...

Targeted Oncology connects oncology professionals with updates on immunotherapy, biomarkers, cancer pathways, and targeted ...

“Dancing molecules,” the promising new treatment for acute spinal cord injuries developed at Northwestern University, has received Orphan Drug Designation from the ... is helping navigate the FDA ...

In March 2025, KRRO-110 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of AATD. KRRO-110 is currently being evaluated in the Phase 1/2a REWRITE clinical ...

Marseille, France Monday, July 21, 2025, 12:00 Hrs [IST] ...

As one FDA initiative works to sustain the momentum of rare disease research, other events threaten to slow progress.

Buried within the One Big Beautiful Bill Act is a provision that could improve or even save the lives of the 30 million ...

Orphan drug designation is intended to support the development and evaluation of new treatments for rare diseases affecting fewer than 200,000 people in the United States.

IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercialization.

"The Orphan Drug Designation granted to Equecabtagene Autoleucel Injection in South Korea marks another significant international regulatory milestone for the product, following its ODD approval ...