Blog
RSS FeedPosted by Michael Wonder on 21 Jan 2020
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 ...
Posted by Michael Wonder on 17 Jan 2020
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 ...
Posted by Michael Wonder on 16 Jan 2020
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 ...
Posted by Michael Wonder on 12 Jan 2020
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 ...
Posted by Michael Wonder on 09 Jan 2020
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 ...
Posted by Michael Wonder on 09 Jan 2020
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 ...
Posted by Michael Wonder on 06 Jan 2020
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 ...
Posted by Michael Wonder on 06 Jan 2020
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 ...
Posted by Michael Wonder on 19 Dec 2019
'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 ...
Posted by Michael Wonder on 18 Dec 2019
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 ...
Posted by Michael Wonder on 13 Dec 2019
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 ...
Posted by Michael Wonder on 12 Dec 2019
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 ...
Posted by Michael Wonder on 11 Dec 2019
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 ...
Posted by Michael Wonder on 10 Dec 2019
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. ...
Posted by Michael Wonder on 03 Dec 2019
EpiPen Jr
2 December 2019 - Due to the critical nature of the ongoing EpiPen Jr shortage, the TGA is allowing one ...