Catherine Skari
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Feb- 2023 -14 FebruaryNews
COMING UP | Share Your Colours For Rare Disease Day 2023
A Few Words About Rare Diseases and the RDD2023 Campaign Rare Disease Day (RDD) is the official international awareness-raising campaign…
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10 FebruaryScientific Literature
Association of Sickle Cell Disease With Severe Maternal Morbidity
Objective: To evaluate the association between sickle cell disease (SCD) and severe maternal morbidity (SMM) in a contemporary cohort of…
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9 FebruaryNews
CLINICAL RESULTS | Roche Reports Positive Data for Crovalimab as Rare Blood Disease Treatment
PNH causes a patient’s red blood cells to break apart, resulting in a range of debilitating symptoms such as anaemia,…
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Jan- 2023 -28 JanuaryScientific Literature
Juggling Between the Cost and Value of New Therapies: Does Science Still Serve Patient Needs?
Thalassaemia International Federation (TIF), representing the united voice of people with thalassaemia and their families globally, has been striving for…
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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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