Novartis firm AveXis gets EC nod for Zolgensma to treat spinal muscular atrophy
The approval allows Zolgensma to treat patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1or patients with
Skyline Therapeutics has announced that the US Food and Drug Administration (FDA) granted orphan drug designation (ODD) to its gene therapy, SKG1108, for the treatment of Retinitis Pigmentosa (RP), an inherited retinal disease.
Can-Fite BioPharma, a biotechnology company advancing a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, today announced it has filed a pre-Investigational New
Four metastatic pancreatic cancer patients have been treated with PD-L1 t-haNK and N-803 under single patient INDs, with two patients on treatment for an evaluable period. One patient reported
FDA Fast Track is a program designed to facilitate and expedite the development and review of a new drugs that demonstrates the potential to address an unmet medical
GA is a progressive, irreversible retinal disease that occurs in patients with age-related macular degeneration (AMD) for which there is no current treatment. Together, with Boehringer Ingelheim’s expertise