Catherine Skari
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Jan- 2023 -5 JanuaryNews
CLINICAL UPDATE | Graphite’s Hopes For Sickle Cell Cure Blunted After First Patient Dosed Experiences Serious Event
The company cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) as part of…
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1 JanuaryScientific Literature
Overall and Complication‐free Survival in a Large Cohort of Patients With β‐Thalassemia Major Followed Over 50 Years
We report data on survival and complications for a longitudinal cohort of 709 transfusion-dependent β-thalassemia major patients (51.1% males) born between…
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Dec- 2022 -16 DecemberNews
RARE ANAEMIAS | Shining a Spotlight on Rare Anaemias’ Education and Advocacy with RAIN
Recognising the absence of a global umbrella organisation for rare anaemias to advocate and safeguard the rights of patients in…
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14 DecemberNews
CLINICAL UPDATE | Novartis Builds Case for Iptacopan as Oral Rival to PNH Injectables
On December 8, the Swiss pharma group announced that the top-line results of the APPOINT-PNH clinical trial showed that iptacopan…
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14 DecemberNews
SCIENCE NEWS | Research On Rare Genetic Disease Sheds Light On A Common Head And Neck Cancer
Patients with the rare genetic disorder Fanconi anemia lack certain elements of this repair system, making their cells incapable…
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12 DecemberNews
PROFILE | After Major Advances In Hematology, Dr David Bodine Retires
When David M. Bodine, Ph.D., arrived at the US National Institutes of Health (NIH) for a postdoctoral fellowship in Arthur Nienhuis’s…
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12 DecemberNews
CLINICAL NEWS | First Cohort of Patients Dosed In Phase 2 Study Of KP104 In PNH
KP104 is a first-in-class, bifunctional biologic designed to modulate complement activity through selective targeting of key intervention points in the…
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12 DecemberNews
DIAGNOSTICS | FDA Clears Baebies Disposable Enzyme Deficiency G6PD Test
Using a 50 µL blood sample (one drop of blood), FINDER delivers results approximately 15 minutes after sample introduction.…
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Nov- 2022 -25 NovemberNews
DRUGS | EMA Starts Pilot To Collect Information On Ongoing/Potential Shortages
To help address this issue, the European Medicines Agency (EMA) is starting a pilot to collect information on ongoing…
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21 NovemberScientific Literature
Emergent Treatments for β-Thalassemia and Orphan Drug Legislations
Highlights • β-THAL is one of the most prevalent blood disorders in some regions of the world. • However, given its prevalence,…
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