Zynteglo™ is a gene therapy indicated for the treatment of transfusion-dependent beta (β)-thalassemia...
Fulcrum launches Phase IIb trial of losmapimod to treat FSHD
Fulcrum Therapeutics has started a Phase IIb clinical trial of losmapimod in patients suffering from facioscapulohumeral muscular dystrophy (FSHD).
ProQR to study new drug candidate for retinitis pigmentosa
ProQR Therapeutics has announced plans to conduct a Phase I/II clinical trial to evaluate QR-1123 for the treatment of patients with autosomal dominant retinitis pigmentosa (adRP).
Arrowhead initiates Phase II/III trial of RNAi therapeutic ARO-AAT
Arrowhead Pharmaceuticals has initiated a potentially pivotal Phase II/III clinical trial of ARO-AAT, its second generation subcutaneously administered RNA interference (RNAi) therapeutic.
Translate Bio’s mRNA candidate produces mixed results in CF trial
Translate Bio has reported positive interim data from a Phase I/II clinical trial of its mRNA candidate MRT5005 for the treatment of cystic fibrosis (CF) patients.
Zynteglo for the Treatment of Transfusion-Dependent Beta-thalassemia
Zynteglo™ is a gene therapy indicated for the treatment of transfusion-dependent beta (β)-thalassemia (TDT) in patients aged 12 years and older.
Zolgensma for the Treatment of Spinal Muscular Atrophy
Zolgensma® (onasemnogene abeparvovec-xioi) is a gene replacement therapy indicated for the treatment of spinal muscular atrophy (SMA) in paediatric patients.
Lamzede (velmanase alfa) to Treat Alpha-Mannosidosis
Lamzede® (velmanase alfa) is one of the first enzyme replacement therapies indicated for the treatment of non-neurological symptoms of patients with mild-to-moderate alpha‑mannosidosis.
Crysvita (burosumab-twza) for Treating X–Linked Hypophosphatemia
Crysvita® (burosumab-twza) is a fibroblast growth factor 23 (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adults and children.
Gamifant (emapalumab-lzsg) for Primary Haemophagocytic Lymphohistiocytosis
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ) blocking antibody indicated for the treatment of primary haemophagocytic lymphohistiocytosis (HLH) in paediatric and adult patients.