The FDA has granted Orphan Drug designation to riliprubart for the investigational treatment of AMR in solid organ transplantation.

CURE connects oncology patients, survivors, and caregivers with expert guidance, cancer updates, treatment education, and ...

“Professor Mellerio brings an important clinical voice to the NETHERTON NOW series,” said Denise Carter, Co-Founder and Chief Operating Officer of Quoin Pharmaceuticals. “Her experience treating ...

Shares of Neuren Pharmaceuticals Ltd (ASX:NEU) jumped over 10% on Thursday after the company announced a key development in ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational drug, riliprubart, for the ...

The U.S. FDA has granted orphan drug designation for Eli Lilly's olomorasib for treatment of KRAS G12C-mutant non-small cell ...

(TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS ...

UC San Diego scientists have created a gene therapy that goes beyond masking Alzheimer’s symptoms—it may actually restore ...

The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines ...

Congenital adrenal hyperplasia (CAH) is a rare autosomal recessive disease1 with an estimated global incidence of approximately 1 in 14,000–18,000 live births2A phase II trial has recently been ...

Bristol-Myers Squibb has secured a pivotal FDA orphan drug designation for its sickle cell disease treatment, signaling a ...