A new distributional cost-effectiveness analysis of gene therapy versus standard-of-care for sickle cell disease (SCD) found that while gene therapy is cost-ineffective by conventional measures, it can be an equitable therapeutic ...
May 29, 2023
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There is growing evidence that a gene variant that reduces the plasticity of the nervous system also modulates responses to treatments for mood and anxiety disorders. In this case, patients with posttraumatic stress disorder, ...
Jun 11, 2013
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A prominent protein activated by inflammation is the key instigator that converts glioblastoma multiforme cells to their most aggressive, untreatable form and promotes resistance to radiation therapy, an international team ...
Aug 29, 2013
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(Medical Xpress)—Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, ...
Dec 9, 2013
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In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and ...
Apr 21, 2015
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Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, ...
Apr 22, 2024
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A single session of a gene therapy developed by the Universitat Autonoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every ...
Jul 2, 2013
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Encouraging safety and efficacy data on novel and emerging therapies presented at the 54th Annual Meeting of the American Society of Hematology (ASH) signal an important step forward in the development of treatment strategies ...
Dec 9, 2012
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For the last several decades, scientists worldwide have been seeking to harness the power of stem cells to develop therapies for human diseases and conditions. At UCLA's Broad Stem Cell Research Center, the potential to bring ...
Jun 14, 2016
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The U.S. Food and Drug Administration on Thursday approved the drug Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).
Jun 26, 2023
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