Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.
Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders.
Pharming has submitted its oral phosphoinositide 3-kinase delta (PI3Kδ) inhibitor leniolisib for approval in the EU as a treatment for activated PI3K delta syndrome (APDS), an ultra-rare di
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRe
Sanofi and Regeneron have claimed the first approval in the US for a drug for prurigo nodularis, after the FDA gave a green light to Dupixent as a treatment for adults with the rare skin di
After a torrid couple of years, Biogen can be forgiven for making the most of new data on its tofersen drug for amyotrophic lateral sclerosis (ALS) – with an elevated status among its pipel