Synopsis: SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys with this devastating and…
Rare
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RegulatoryU.S FDAVertex
Vertex Announces U.S. FDA Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 12 to 24 months
by adminby adminSummary: Orkambi was first approved in 2015 in the US and is now available in more than 30 countries. Vertex Pharmaceuticals Incorporated…
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Synopsis : Rare viral haemorrhagic disease similar to Ebola surface in the East of England. Two people in the UK have been…
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AgrochemicalsGlobal MarketPharma Science & Research
Orchard Therapeutics Announces Historic Agreement Making Libmeldy Available by NHS England for MLD Patients
by adminby adminSummary : First-ever lentiviral hematopoietic stem cell gene therapy approved for reimbursement by NHS England Two children with early-onset MLD recently treated…
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Agrochemicals
EUSA Pharma will be Acquired by Italian Firm Recordati for €750 Million Will Boost Rare Disease Offering
by adminby adminItalian drugmaker Recordati has agreed on a price for six-year-old EUSA Pharma. The Milan-based firm will pay €750m ($845 million) for EUSA’s…
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Ropeginterferon
FDA Approves Ropeginterferon Alfa-2b-njft (Besremi) Treatment for Rare Blood Disease Polycythemia Vera (PV).
by adminby adminSUMMARY: PharmaEssentia Corporation a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in…
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Agrochemicals
Kerala’s Native herb ‘Manathakkali’ (Solanum nigrum) Approved for Liver Cancer, Research gets US FDA Nod.
by adminby adminKEYPOINTS: ‘Manathakkali’ (Black nightshade or Solanum nigrum), a berry-bearing shrub found in the backyard, is traditionally known for its medicinal value. Two scientists…
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Active Pharmaceutical Ingredients (API)Ataluren
ANVISA grants expanded Approval to PTC’s Translarna label to include Ambulatory Patients as young as 2 years old in Brazil.
by adminby adminKeypoints: PTC Therapeutics, Inc. announced that the Brazilian National Health Surveillance Agency has approved the expansion of the indication of Translarna (Ataluren)…