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Multiple myeloma treatment granted Orphan Drug Designation
Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma.
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Orphan Drugs
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Multiple myeloma treatment receives Orphan Drug Designation
Orphan Drug Designation has been granted to the CT053 anti-BCMA CAR-T programme for multiple myeloma treatment.
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FDA grants Orphan Drug Designation to DYN101 treatment
A treatment for Centronuclear Myopathies has received Orphan Drug Designation from the FDA and will begin Phase I/II trials.
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FDA grants Orphan Drug Designation for narcolepsy treatment
The FDA has given the XWL-008 compound Orphan Drug Designation for the treatment of narcolepsy.
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FDA approves first gene therapy for paediatric spinal muscular atrophy
The FDA has given approval to AveXis for the first treatment of paediatric spinal muscular atrophy (SMA).
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Eurofins Amatsigroup ǀ Your cDmo partner
Frédéric Gaussens from Eurofins Amatsigroup (CDMO) giving an overview of the services they provide...
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FDA grants orphan drug designation to elamipretide for LHON
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber's hereditary optic neuropathy...
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FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia
The FDA has approved Crysvita, the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets...
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KL1333 receives FDA ODD for mitochondrial diseases
NeuroVive Pharmaceutical AB has been granted Orphan Drug Designation for its project KL1333 for treatment of inherited mitochondrial respiratory chain diseases...
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FDA expands approval of Blincyto for treatment of B-cell precursor ALL
The FDA has granted accelerated approval to Blincyto to treat adults and children with B-cell precursor acute lymphoblastic leukaemia (ALL)...
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FDA approves Lutathera for somatostatin receptor-positive GEP-NETs
The FDA has approved Lutathera for the treatment of a type of cancer that affects the pancreas the gastrointestinal tract...
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Enzyme replacement therapy to treat alpha-mannosidosis
The EMA has recommended granting a marketing authorisation in the EU for Lamzede a long-term enzyme replacement therapy for alpha-mannosidosis...
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EC grants orphan drug designation for Gilteritinib for the treatment of AML
The European Commission has issued Orphan Designation to gilteritinib for the treatment of patients with acute myeloid leukaemia...
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FDA accepts IND for the treatment of mucopolysaccharidosis type IIIA
The FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for the drug candidate SOBI003...
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FDA approves Nucala for Eosinophilic Granulomatosis with Polyangiitis
The FDA has expanded the approved use of Nucala to treat a rare autoimmune disease that causes vasculitis, an inflammation of the wall of blood vessels of the body...
