#thalassaemia

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www.independent.co.uk
7 months ago
Medicine

Adults with rare blood disorder to be offered stem cell transplants

Adults with thalassaemia can now benefit from stem cell transplants to cure their condition, previously only available to children.
New guidelines recommend making stem cell transplants available to eligible adult patients, with over 600 people set to benefit.
Expanding the availability of stem cell transplants to adults with thalassaemia is a significant step forward in improving their quality of life. [ more ]
#CRISPR gene editing
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
www.scientificamerican.com
7 months ago
Health

U.K. Becomes First Country to Approve a CRISPR Disease Treatment

UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
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