News
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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 for rare diseases which takes place on February 28. The…
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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, fatigue, blood clots and kidney disease. The treatment is for…
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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 CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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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 providing improvements in hematologic [blood-related] parameters, treatment with [Enjaymo] also…
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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 potential to address a wide range of hematologic disorders including…
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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 pediatric disease designation for the treatment of severe combined immunodeficiency…
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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 latest development is based on the findings from the head-to-head…
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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 a phase 1/2 trial for SCD. The event is likely to…
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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 a consistent way, and the commonalities between rare anaemias 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 provided “clinically meaningful” increases in haemoglobin levels in patients with…
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