Remove tag als-association
article thumbnail

Actualising the power of antibody-drug conjugates as cancer therapeutics

European Pharmaceutical Review

The antibody is chosen based on its high selectivity and affinity for the tumour-associated antigen and its ability to facilitate internalisation or endocytosis, wherein molecules such as proteins are engulfed by the cell membrane and drawn into the cell. Fu Z, Li S, Han S, et al. Zacharias N, Podust V, Kajihara K, et al.

article thumbnail

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

Trending Sources

article thumbnail

Unconventional Approaches Pharma Is Taking to Engage Patients

PM360

Then onto Tuan Vu, MD, Professor of Neurology, Director, Neuromuscular Division, EMG Laboratories, and ALS Clinic, Department of Neurology University of South Florida, who discussed the global clinical trial. We also put big pull tags on the little pouches that held the items.

Patients 105
article thumbnail

Developing point-of-care CAR T manufacturing

European Pharmaceutical Review

This is especially important due to the two unique toxicities of CAR T cells, namely CRS and the neurology toxicities that subsequently received the term immune effector cell- associated neurotoxicity syndrome (ICANS). This is in addition to its considerable price tag. Grupp SA, Kalos M, Barrett D, et al. Asghar N, et al.