Ads
related to: sickle cell gene therapy- Learn About Treatment
Learn About the Treatment Journey.
Watch the Video to Learn More.
- View Discussion Guides
Download Treatment Brochures,
Discussion Guides, and More.
- Safety and Side Effects
Review the Important Safety
Information & Side Effects.
- Patient FAQs
Get Answers to Common Questions.
Understand the Potential Risks.
- Find an ATC
Find an Authorized Treatment Center
On the Official Patient Website.
- Patient Information
View Important Patient Information.
Understand the Treatment.
- Learn About Treatment
Search results
Results From The WOW.Com Content Network
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]
CRISPR Therapeutics' first therapy is called Casgevy. It treats sickle cell disease (SCD) and beta thalassemia, both rare inherited illnesses that affect the function of a person's red blood cells.
In December 2023, the FDA approved the first gene therapy in the US to treat patients with Sickle Cell Disease (SCD). The FDA approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of SCD. [57]
They include other target indications for povetacicept and suzetrigine, a next-generation nonopioid pain drug (VX-993), a messenger RNA therapy for CF (VX-552), and a therapy targeting myotonic ...
This therapy treats two blood-related disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Casgevy is approved in several regions: the U.S., the U.K., the ...
This is an accepted version of this page This is the latest accepted revision, reviewed on 15 September 2024. Manipulation of an organism's genome For a non-technical introduction to the topic of genetics, see Introduction to genetics. For the song by Orchestral Manoeuvres in the Dark, see Genetic Engineering (song). For the Montreal hardcore band, see Genetic Control. Part of a series on ...
Ads
related to: sickle cell gene therapy