Sickle Cell Disease
TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020
Actualités
il y a 5 jours
TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020
TIF is proudly organising the European Thalassaemia & Sickle Cell Disease Symposium, a joint patients’ and healthcare professionals’ three-day educational event which will take place online on 11 – 13 December…
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
Actualités
il y a 4 semaines
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in…
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
Actualités
il y a 4 semaines
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
Actualités
octobre 7, 2020
Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
Fulcrum Therapeutics, Inc. a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and…
Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Actualités
octobre 2, 2020
Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…
TIF Takes Active Part At The IAPO 9th Global Patients Congress
Actualités
septembre 29, 2020
TIF Takes Active Part At The IAPO 9th Global Patients Congress
The International Alliance of Patients’ Organizations (IAPO) conducted online the 9th Global Patients Congress #GPC2020 on 16-17 September 2020, under the theme »Co-creation of Innovative Healthcare ». The Congress featured a…
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
Actualités
septembre 25, 2020
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…
Coronavirus Disease among Persons with Sickle Cell Disease
Actualités
juillet 13, 2020
Coronavirus Disease among Persons with Sickle Cell Disease
Individuals with sickle cell disease (SCD) are a particularly vulnerable group of patients, with a higher risk of severe complications due to COVID-19 infection than the general public. A recent…
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Actualités
juillet 7, 2020
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe
Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease…
IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
Actualités
juin 30, 2020
IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the…