Catherine Skari

HSCT | Briquilimab Granted Orphan Drug Designation By The European Commission

Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to briquilimab in HCT, as well as rare…

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NEW APPROVAL | Health Canada Grants Approval To Sobi’s PNH Therapy

The treatment is indicated for such patients with insufficient response to, or are not tolerant to, a C5 inhibitor. The…

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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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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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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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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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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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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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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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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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