Ads
related to: sickle cell disease gene therapy- Learn About Treatment
Learn About the Treatment Journey.
Watch the Video to Learn More.
- Safety and Side Effects
Review the Important Safety
Information & Side Effects.
- View Discussion Guides
Download Treatment Brochures,
Discussion Guides, and More.
- Patient Information
View Important Patient Information.
Understand the Treatment.
- Patient FAQs
Get Answers to Common Questions.
Understand the Potential Risks.
- Find an ATC
Find an Authorized Treatment Center
On the Official Patient Website.
- Learn About Treatment
Search results
Results From The WOW.Com Content Network
DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a patient’s ...
Sickle cell is the most common disease to receive approval for gene therapy treatment, following decades of development and years of approvals for therapy for people with rarer conditions.
Sickle cell disease (SCD) is a painful, inherited blood disorder in which the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the ...
Diseases such as sickle cell disease for which a person's normal phenotype or cell function may be restored in cells that have the disease by a normal copy of the gene that is mutated, may be a good candidate for gene therapy treatment. The risks and benefits related to gene therapy for sickle cell disease are not known. [191]
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
Ads
related to: sickle cell disease gene therapy