children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various ...

In a report released today, Brian Abrahams from RBC Capital maintained a Buy rating on PTC Therapeutics (PTCT – Research Report), with a price ...

Vabysmo PFS is the first and only prefilled syringe containing a bispecific antibody, offering a convenient alternative to curr ...

Vabysmo PFS is the first and only prefilled syringe containing a bispecific antibody, offering a convenient alternative to currently available Vabysmo vialsVabysmo has demonstrated rapid and robust vi ...

It was also under development for the treatment of Rett syndrome, Melas syndrome, Leber’s hereditary optic neuropathy and mitochondrial diseases such as Pearson syndrome, a form of mitochondrial ...

SAN ANTONIO -- The addition of palbociclib (Ibrance) to current standard-of-care therapy following induction chemotherapy ...

Progression-free survival was superior with first-line maintenance of palbociclib in HR-positive, HER2-positive metastatic ...

Crucially for the Netherlands-based biopharma company, the FDA has said that the composite Unified Huntington's Disease ...

The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies ...

Relation Therapeutics Ltd. has delivered the first commercial validation of its combined wet lab/in silico platform for homing in on disease-related genes in the thick of the genome, signing two ...

Qure's AMT-130 shows promising results for Huntington’s disease. Learn why QURE stock faces commercialization challenges ...

Raymond James and Cantor both released bullish notes on uniQure (NASDAQ:QURE) Tuesday in the wake of news that the company ...