Mum says 'miracle' drug saved ill baby Kaan Hunter's life

21 January 2020 - A baby who was given only four months to live after being diagnosed with an extremely ...

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Fortress Biotech announces rare paediatric disease designation for CUTX-101 for the treatment of Menkes disease

16 January 2020 - Cyprium Therapeutics, a Fortress partner company, on track to begin submitting rolling new drug application for CUTX-101 ...

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Asfotase alfa in hypophosphatasia in children and adolescents: survival advantage for small children

15 January 2020 - Weak evidence does not allow a specific statement to be made about the extent of the added ...

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Sanofi secures expanded European approval for Toujeo in type 1 diabetes

10 January 2020 - Sanofi has revealed that the European Commission has expanded the existing label for Toujeo (insulin glargine ...

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U.S. FDA approves supplemental new drug application for expanded indication of Mycamine (micafungin for injection) for the treatment of invasive candidiasis in paediatric patients less than 4 months of age

8 January 2020 - Astellas Pharma today announced that the U.S. FDA has approved its supplemental new drug application for ...

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Aruvant receives FDA rare paediatric disease designation for ARU-1801 for the treatment of sickle cell disease

8 January 2020 - ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and ...

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FDA approves Fiasp for treatment of children with diabetes

6 January 2020 - Novo Nordisk today announced that the U.S. FDA has approved Fiasp (insulin aspart injection) 100 u/mL for ...

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Canadian parents of babies with rare deadly disease look to Novartis treatment lottery

5 January 2020 - Canadian babies with a rare and often fatal muscle-wasting disease may soon have access to a ...

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'Healthy lottery' fears as Novartis plans to give away world’s most expensive drug

19 December 2019 - Novartis aims to give away 100 doses of its $US2.1 million-per-patient ($3.05 million) Zolgensma for spinal ...

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Chondrial Therapeutics announces dosing of first patients in Phase 1 clinical program of CTI-1601 for treatment of Friedreich’s ataxia; CTI-1601 granted rare pediatric disease designation and fast track designation by U.S. FDA

18 December 2019 - Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, with an initial ...

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Y-mAbs’ GD2-GD3 vaccine granted rare paediatric disease designation

12 December 2019 - Y-mAbs Therapeutics today announced that its GD2-GD3 vaccine has been granted a rare paediatric disease designation ...

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FDA issues draft guidance to foster oncology product development for paediatric populations

12 December 2019 - Today, the U.S. FDA issued a draft guidance document, “FDARA Implementation Guidance for Pediatric Studies of ...

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Vertex announces European Commission approval for Kalydeco (ivacaftor) in infants with cystic fibrosis ages 6 months to less than 12 months with certain mutations in the CFTR gene

10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...

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Pickering mom fights for life-changing CF medicine to come to Canada

10 December 2019 - Stephanie Stavros feels like she's 'screaming into an abyss' as her life slips away. ...

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EpiPen Jr

2 December 2019 - Due to the critical nature of the ongoing EpiPen Jr shortage, the TGA is allowing one ...

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