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beta thalassaemia
This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.
Bristol Myers Squibb’s plaque psoriasis treatment recommended by CHMP
The company also received a recommendation from the CHMP this month for Reblozyl (luspatercept) as a treatment for adult patients with anaemia associated with non-transfusion-dependent beta thalassaemia. ... Noah Berkowitz, senior vice president,
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bluebird bio sells second FDA priority review voucher for $95m
bluebird bio received two PRVs upon the FDA approvals of Zynteglo (betibeglogene autotemcel) for the treatment of beta-thalassaemia in adult and paediatric patients requiring regular red blood cell transfusions, and
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Jasper Therapeutics’ briquilimab given orphan drug designation in EU
The company also announced positive results earlier this month from a phase 1/2 trial of briquilimab as a conditioning treatment in patients with sickle cell disease (SCD) and beta thalassaemia
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FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies
It was found that both patients had a specific genotype known as alpha thalassaemia trait, which has now been added to exclusion criteria for ongoing studies. ... The prior month, bluebird’s Zynteglo (betibeglogene autotemcel) was also approved by the
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bluebird bio sells Rare Pediatric Disease Priority Review Voucher
bluebird bio received two PRVs upon the FDA approvals of Zynteglo (betibeglogene autotemcel) for the treatment of beta-thalassaemia (beta-thal) in adult and paediatric patients requiring regular red blood cell ... Beta-thal is a rare genetic blood
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BMS’s Reblozyl meets primary endpoint in myelodysplastic syndromes phase 3 trial
The treatment is currently approved in the US for anaemia in adult patients with beta thalassaemia who require regular red blood cell transfusions, as well as for the treatment of anaemia
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Case-study for thalassaemia: Dimitri Azzopardi
An in-depth look at beta thalassaemia for Rare Disease Day. Beta thalassaemia major is a serious, lifelong genetic blood disorder. ... At least 75, 000 children are born. with thalassaemia worldwide every year, of which 10, 000 have the debilitating form
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20 for 2020 – Five pharma companies to watch
treatment ozanimod and myelodysplastic syndromes/beta thalassaemia candidate luspatercept – to deliver on their potential.
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