Decibel Therapeutics receives orphan drug and rare paediatric disease designations for DB-OTO for the treatment of otoferlin related congenital hearing loss

9 September 2021 - Decibel Therapeutics today announced that the U.S. FDA has granted both orphan drug designation and rare paediatric ...

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NICE recommends treatment for people with juvenile idiopathic arthritis

9 September 2021 - NICE has today published final draft guidance which recommends tofacitinib (Xeljanz; Pfizer) as an option for treating ...

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U.S. FDA accepts for priority review supplemental new drug application for Oxbryta (voxelotor) for the treatment of sickle cell disease in children ages 4 to 11

7 September 2021 - New drug application for Oxbryta dispersible tablets also accepted for priority review. ...

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COVID-19 vaccine: Spikevax (elasomeran)

4 September 2021 - On 3 September 2021, the TGA granted provisional approval to Moderna Australia for the use of ...

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NICE recommends Novartis’ Cosentyx for children with severe psoriasis

3 September 2021 - NICE has recommended Novartis’ interleukin-17A inhibitor Cosentyx (secukinumab) for the treatment of children and young people ...

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Ultomiris approved in the EU for children and adolescents with paroxysmal nocturnal haemoglobinuria

3 September 2021 - Approval based on interim results from Phase 3 trial demonstrating complete terminal complement inhibition with reduced ...

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UCB announces Briviact (brivaracetam) now FDA approved to treat partial-onset seizures in paediatric patients one month of age and older

30 August 2021 - Tablets, oral solution and intravenous dosage forms of Briviact (brivaracetam) now FDA approved as both monotherapy or ...

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Evrysdi (risdiplam) receives CADTH reimbursement recommendation for some patients with spinal muscular atrophy

30 August 2021 - Roche Canada today announced that the CADTH Canadian Drug Expert Committee has issued its final recommendation ...

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Moderna COVID-19 vaccine gets Health Canada approval for kids 12+

27 August 2021 - As the Delta variant rages through unvaccinated populations in Canada ahead of the back-to-school season, children ...

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ATAGI recommendations on the use of COVID-19 vaccines in all young adolescents in Australia

27 August 2021 - Vaccination against COVID-19 is recommended for all individuals from 12 years of age, extending the current ...

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European Commission approves BioMarin's Voxzogo (vosoritide) for the treatment of children with achondroplasia from age 2 until growth plates close

27 August 2021 - First medicine approved to treat children with achondroplasia in Europe ...

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Vertex Pharma gets approval for cystic fibrosis drug for infants in Canada

25 August 2021 - Vertex Pharmaceuticals  was given approval to market its drug ivacaftor that treats children as young as ...

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Ascendis Pharma announces U.S. FDA approval of Skytrofa (lonapegsomatropin-tcgd), the first once weekly treatment for paediatric growth hormone deficiency

25 August 2021 - Availability in the U.S. expected shortly supported by a full suite of patient support programs. ...

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Miravo Healthcare announces receipt of Health Canada Notice of Compliance for the paediatric use of Blexten

12 August 2021 - Blexten Label Expanded to Include Ages as Young as 4 Years Old. ...

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Moderna COVID-19 vaccine approved by MHRA in 12-17 year olds

17 August 2021 - The UK regulator has confirmed the vaccine is safe and effective in this age group. ...

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