Children’s Hospital of Philadelphia (CHOP) was recently named a Qualified Treatment Center (QTC) for Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Lenmeldy has the potential to stop or slow disease progression with a single cell-based treatment.
CHOP’s Leukodystrophy Center is one of only a handful of centers in the nation that brings together a multidisciplinary group of caregivers to provide cutting-edge, comprehensive diagnostic testing, clinical care, and treatment to infants and children with inherited white matter disease. The center has one of the most experienced teams of experts in the world when it comes to diagnosing and treating MLD, a rare, autosomal recessive lysosomal storage disorder that results in progressive neurologic disability.
Patients receiving Lenmeldy will be treated by CHOP’s Cellular Therapy and Transplant Section in partnership with the Leukodystrophy Center. CHOP’s Cell Therapy and Transplant program is one of the most experienced gene therapy programs in the world, and it is the only program in the United States to be a Qualified Treatment Center for all six FDA-approved stem cell-based gene therapies for genetic blood and metabolic disorders (Zynteglo, Skysona, Lyfgenia, Casgevy thalassemia, Casgevy SCD and Lenmeldy). CHOP was directly involved in the FDA approval process for many of them.
CHOP continues to be a global leader in cell and gene therapies. The Cell Based Therapy Laboratory, which has been processing cells for transplants for decades, has taken on an increasing number of experimental cellular therapy protocols since 2010. CHOP is also home to the Raymond G. Perelman Center for Cellular and Molecular Therapeutics Clinical Vector Core, which utilizes a vast array of state-of-the-art technology for preclinical and clinical vector production and characterization used in the development of gene therapies.
To learn more about this treatment option, contact CHOP’s Leukodystrophy Center at LCE@chop.edu.
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Children’s Hospital of Philadelphia (CHOP) was recently named a Qualified Treatment Center (QTC) for Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Lenmeldy has the potential to stop or slow disease progression with a single cell-based treatment.
CHOP’s Leukodystrophy Center is one of only a handful of centers in the nation that brings together a multidisciplinary group of caregivers to provide cutting-edge, comprehensive diagnostic testing, clinical care, and treatment to infants and children with inherited white matter disease. The center has one of the most experienced teams of experts in the world when it comes to diagnosing and treating MLD, a rare, autosomal recessive lysosomal storage disorder that results in progressive neurologic disability.
Patients receiving Lenmeldy will be treated by CHOP’s Cellular Therapy and Transplant Section in partnership with the Leukodystrophy Center. CHOP’s Cell Therapy and Transplant program is one of the most experienced gene therapy programs in the world, and it is the only program in the United States to be a Qualified Treatment Center for all six FDA-approved stem cell-based gene therapies for genetic blood and metabolic disorders (Zynteglo, Skysona, Lyfgenia, Casgevy thalassemia, Casgevy SCD and Lenmeldy). CHOP was directly involved in the FDA approval process for many of them.
CHOP continues to be a global leader in cell and gene therapies. The Cell Based Therapy Laboratory, which has been processing cells for transplants for decades, has taken on an increasing number of experimental cellular therapy protocols since 2010. CHOP is also home to the Raymond G. Perelman Center for Cellular and Molecular Therapeutics Clinical Vector Core, which utilizes a vast array of state-of-the-art technology for preclinical and clinical vector production and characterization used in the development of gene therapies.
To learn more about this treatment option, contact CHOP’s Leukodystrophy Center at LCE@chop.edu.
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Leukodystrophy Center
The Leukodystrophy Center at CHOP provides care, diagnosis and advanced treatments to kids with inherited white matter diseases.
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Through cutting-edge gene therapy research, CHOP is finding novel ways to help children with neurological disorders.
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