News
-
NEW REVIEW | Treatment Landscape Of Rare Anaemias Is Shifting Rapidly
Writing in Frontiers in Medicine, the authors began by reviewing congenital and acquired forms of anaemia. Those include sickle cell disease (SCD) and hemolytic anemias (CHAs), both congenital, and autoimmune hemolytic…
Read More » -
EMERGENCY APPEAL | TIF Rallies to Provide Earthquake Relief in Turkey and Syria
Among these people, the Thalassaemia International Federation (TIF) estimates that at least 2,000 patients with thalassaemia have been affected, 70% of whom are children and adolescents under the age of…
Read More » -
TOP STORY | EU Approves Luspatercept for Anemia in Patients with Non–Transfusion-Dependent β-Thalassaemia
The approval was based on findings from the phase 2 BEYOND trial, which demonstrated that 77.1% of patients treated with luspatercept (n = 74/96) experienced a mean haemoglobin (Hb) increase…
Read More » -
TIF2023 | The Top International Conference On Thalassaemia Returns This November
The Conference, jointly organised by TIF, the Malaysian Haematology Society, and the Federation of Malaysia Thalassaemia Societies, will feature two parallel programmes – a Scientific Programme for Healthcare Professionals and…
Read More » -
SICKLE CELL DISEASE | Hydroxyurea Use Linked To Higher Rates Of Spleen Removal
The study’s findings also indicated that SCD children treated with hydroxyurea underwent splenectomy surgery at a significantly younger age compared with those not given the oral medication. That result came…
Read More » -
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…
Read More » -
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…
Read More » -
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…
Read More » -
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…
Read More » -
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…
Read More »