Catherine Skari
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Jan- 2023 -26 JanuaryNews
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a…
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23 JanuaryNews
COLD AGGLUTININ DISEASE | Enjaymo Improves Life Quality And Lessens Fatigue, According to Phase 3 Trial Results
These findings “further augment the primary efficacy outcomes of the placebo-controlled Phase 3 CADENZA study, demonstrating that in addition to…
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23 JanuaryNews
IRON CONTROL | Disc Medicine And Mabwell Sign Deal For Licence To Antibodies Portfolio
Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023. MWTX-003 has the…
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11 JanuaryNews
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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10 JanuaryNews
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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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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