SCD
-
News
SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017
Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer…
Read More » -
Scientific News
WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and…
Read More » -
Scientific News
Stroke Risk Rises in People with Sickle Cell Disease Despite Treatment Guidelines
A new study reveals that stroke rates are continuing to rise among both adults and children living with sickle cell disease (SCD), despite established standards of care aimed at reducing…
Read More » -
News
GENE THERAPY | FDA Decision on Lovo-cel for SCD Now Expected by Year’s End
The regulatory agency has agreed to review an application from lovo-cel’s developer, Bluebird Bio, requesting the gene therapy’s approval for SCD patients ages 12 and older. Moreover, the FDA has given the application priority…
Read More » -
News
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…
Read More » -
News
NEW HOPE FOR THALASSAEMIA/SCD | UK Trial Of Lab Grown Red Blood Cells Begins
The Restore trial, led jointly by NHS Blood and Transplant and the University of Bristol, is studying the lifespan of the lab grown cells compared with infusions of standard red blood…
Read More » -
News
SICKLE CELL DISEASE | African Health Ministers Launch Drive to Curb SCD’s Toll
This effort, backed by the African Regional Office of the World Health Organization (WHO), aims to curb SCD΄s toll in a region that has historically been hit hard by this…
Read More » -
Clinical News
Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease
Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of…
Read More » -
News
What’s New In Clinical Trials For Thalassaemia/Sickle Cell Disease Drugs And Therapies?
Many pharmaceutical companies are making remarkable progress in their research into candidate treatments for these diseases and 2022 is expected to be yet another eventful year for haemoglobin disorders! Check…
Read More » -
Clinical News
SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic…
Read More »