Miss Zomboko
JF-Expert Member
- May 18, 2014
- 4,502
- 9,281
Tiba mpya inayokaguliwa na Kamati huru ya Ushauri wa Chakula na Dawa ya Marekani inaweza kuwa Tiba ya kwanza ya Ugonjwa wa Selimundu (Sicklecell) Duniani.
Ikiidhinishwa, Dawa hiyo inayoitwa Exacel iliyotengenezwa na Kampuni ya Vertex Pharmaceuticals ya Boston na Kampuni ya Uswisi ya CRISPR Therapeutics itafanya Kazi katika Mfumo wa 'Genetics' kwa kufanya urekebishaji wa DNA.
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A new treatment under review by the US Food and Drug Administration’s independent advisory committee could cure sickle cell disease.
If approved, the new treatment called exa-cel, made by Boston-based Vertex Pharmaceuticals and the Swiss company CRISPR Therapeutics, will be the first FDA-approved treatment that uses genetic modification called CRISPR.
CRISPR is a technology that researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop.
Up until now, the only treatment for sickle cell disease has been stem cell or bone marrow transplant. In Kenya, the first bone marrow transplant procedure was carried out last October.
Although experts said it opened up the treatment option for people living with sickle cell in the country, FDA states that stem cells transplants are risky and may not work and sometimes a transplant can kill a patient. The agency further indicates that fewer than 20 perc ent of patients have an appropriately matched donor.
The new exa-cel treatment under FDA’s consideration can use the patient’s own stem cells. Doctors would alter them with CRISPR to fix the genetic problems that cause sickle cell, and then the altered stem cells are given back to the patient in a one-time infusion.
Dr Stephanie Krogmeier, vice-president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel that the treatment was considered safeand it had a “highly positive benefit risk for patients with severe sickle cell disease,”
Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshaped red blood cells block normal circulation and can cause moderate to severe pain.
It’s the number one reason patients with sickle cell go to the emergency room or are hospitalised. Before the treatment, patients experienced about four of these painful crises a year, resulting in about two weeks in the hospital.
Ikiidhinishwa, Dawa hiyo inayoitwa Exacel iliyotengenezwa na Kampuni ya Vertex Pharmaceuticals ya Boston na Kampuni ya Uswisi ya CRISPR Therapeutics itafanya Kazi katika Mfumo wa 'Genetics' kwa kufanya urekebishaji wa DNA.
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A new treatment under review by the US Food and Drug Administration’s independent advisory committee could cure sickle cell disease.
If approved, the new treatment called exa-cel, made by Boston-based Vertex Pharmaceuticals and the Swiss company CRISPR Therapeutics, will be the first FDA-approved treatment that uses genetic modification called CRISPR.
CRISPR is a technology that researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop.
Up until now, the only treatment for sickle cell disease has been stem cell or bone marrow transplant. In Kenya, the first bone marrow transplant procedure was carried out last October.
Although experts said it opened up the treatment option for people living with sickle cell in the country, FDA states that stem cells transplants are risky and may not work and sometimes a transplant can kill a patient. The agency further indicates that fewer than 20 perc ent of patients have an appropriately matched donor.
The new exa-cel treatment under FDA’s consideration can use the patient’s own stem cells. Doctors would alter them with CRISPR to fix the genetic problems that cause sickle cell, and then the altered stem cells are given back to the patient in a one-time infusion.
Dr Stephanie Krogmeier, vice-president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel that the treatment was considered safeand it had a “highly positive benefit risk for patients with severe sickle cell disease,”
Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshaped red blood cells block normal circulation and can cause moderate to severe pain.
It’s the number one reason patients with sickle cell go to the emergency room or are hospitalised. Before the treatment, patients experienced about four of these painful crises a year, resulting in about two weeks in the hospital.
