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Prasad Hospitals  >>  Inborn Errors of Metabolism.

Inborn Errors of Metabolism.

Inborn errors of metabolism (IEM) are a class of genetic diseases. The majority are due to defects of single genes that code for enzymes that facilitate the conversion of various substrates into products. In most of the disorders, the accumulation of substances, which are toxic or interfere with normal function arises the problems. Inborn errors of metabolism are also called congenital metabolic diseases or inherited metabolic disorders.

  • icon Lysosomal storage disorders
  • icon Galactosemia
  • icon Maple sugar urine disease (MSUD)
  • icon Phenylketonuria (PKU)
  • icon Adrenoleukodystrophy
  • icon Metachromatic Leukodystrophy
  • icon Globoid Cell Leukodystrophy
  • icon Tay-Sachs Disease
  • icon Sandhoff Disease
  • icon Wolman Disease
  • icon I-Cell Disease
  • icon Sanfilippo Syndrome
  • icon GM1 Gangliosidosis

Symptoms of Inborn Errors of Metabolism:

  • icon Recurrent episodes of vomiting, ataxia, seizures, lethargy, coma or a mix of the above.
  • icon Skeletal abnormalities and abnormalities of the hair or skin.
  • icon Poor feeding, failure to thrive.
  • icon A dilated heart condition, jaundice, and liver dysfunction.
  • icon Developmental delay, occasionally with loss of milestones.
  • icon Ataxia, hypotonia or hypertonia and visual and auditory disturbances.

How can Stem cells help in IEM:

Allogeneic HSCT has largely contributed to the improved quality of life of many children who are affected by IEM.

What Sets Us Apart:

  • icon Prasad Research Foundation along with Prasad Hospitals offers the best stem cell and gene therapy in Hyderabad, India.
  • icon Advisory board of pediatricians, geneticist and researchers.
  • icon India’s best research and practice-oriented stem-cell research practice.

Stem Cell And Gene Therapy in Hyderabad

The purpose of this treatment is to introduce hematopoietic cells obtained from a normal donor containing an enzyme that can avoid substances that have accumulated in the body of people suffering from storage diseases. Hematopoietic cells can be acquired from Peripheral blood i.e., blood circulates the body’s blood vessels; bone marrow; blood is drawn from in between giving birth to the baby and the umbilical cord is cut.

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