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ACMG and GMDI Develop First Guidelines for Medical and Dietary Treatment of PKU

The American College of Medical Genetics (ACMG) and the Genomics and Genetic Metabolic Dietitians International (GMDI) have developed the first-ever specific guidelines for medical and dietary treatment of phenylketonuria (PKU). The guidelines have been cited as “a tremendous step forward in ensuring that all patients in the US and Canada receive the highest quality of care and treatment in the management of their PKU” by the National PKU Alliance and Canadian PKU and Allied Disorders.

The work groups who prepared the documents drew together PKU medical experts from the US and Canada. Key recommendations include the following, among others:

  • Treatment of PKU is lifelong with the goal of maintaining blood PHE levels in the range of 120-360 umol/l (2-6 mg/dl) in patients of all ages for life.
  • Patients treated within the early weeks of life with initial good metabolic control, but who lose that control in later childhood or as an adult, may experience both reversible and irreversible neuropsychiatric consequences.
  • Medical foods (formula and foods modified to be low in protein) are medically necessary for people living with PKU and should be regarded as medications.
  • Any combination of therapies (medical foods, Kuvan, etc.) that improve a patient’s blood PHE levels is appropriate and should be individualized.
  • Reduction of blood PHE, increase in PHE tolerance, or improvement in clinical symptoms of PKU are all valid indications to continue a particular therapy.
  • Genetic counseling should be provided as an ongoing process for individuals with PKU and their families.

The complete medical and dietary guidelines are published on the National PKU Alliance website.  

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The American College of Medical Genetics (ACMG) and the Genomics and Genetic Metabolic Dietitians International (GMDI) have developed the first-ever specific guidelines for medical and dietary treatment of phenylketonuria (PKU). The guidelines have been cited as “a tremendous step forward in ensuring that all patients in the US and Canada receive the highest quality of care and treatment in the management of their PKU” by the National PKU Alliance and Canadian PKU and Allied Disorders.

The work groups who prepared the documents drew together PKU medical experts from the US and Canada. Key recommendations include the following, among others:

  • Treatment of PKU is lifelong with the goal of maintaining blood PHE levels in the range of 120-360 umol/l (2-6 mg/dl) in patients of all ages for life.
  • Patients treated within the early weeks of life with initial good metabolic control, but who lose that control in later childhood or as an adult, may experience both reversible and irreversible neuropsychiatric consequences.
  • Medical foods (formula and foods modified to be low in protein) are medically necessary for people living with PKU and should be regarded as medications.
  • Any combination of therapies (medical foods, Kuvan, etc.) that improve a patient’s blood PHE levels is appropriate and should be individualized.
  • Reduction of blood PHE, increase in PHE tolerance, or improvement in clinical symptoms of PKU are all valid indications to continue a particular therapy.
  • Genetic counseling should be provided as an ongoing process for individuals with PKU and their families.

The complete medical and dietary guidelines are published on the National PKU Alliance website.  

The American College of Medical Genetics (ACMG) and the Genomics and Genetic Metabolic Dietitians International (GMDI) have developed the first-ever specific guidelines for medical and dietary treatment of phenylketonuria (PKU). The guidelines have been cited as “a tremendous step forward in ensuring that all patients in the US and Canada receive the highest quality of care and treatment in the management of their PKU” by the National PKU Alliance and Canadian PKU and Allied Disorders.

The work groups who prepared the documents drew together PKU medical experts from the US and Canada. Key recommendations include the following, among others:

  • Treatment of PKU is lifelong with the goal of maintaining blood PHE levels in the range of 120-360 umol/l (2-6 mg/dl) in patients of all ages for life.
  • Patients treated within the early weeks of life with initial good metabolic control, but who lose that control in later childhood or as an adult, may experience both reversible and irreversible neuropsychiatric consequences.
  • Medical foods (formula and foods modified to be low in protein) are medically necessary for people living with PKU and should be regarded as medications.
  • Any combination of therapies (medical foods, Kuvan, etc.) that improve a patient’s blood PHE levels is appropriate and should be individualized.
  • Reduction of blood PHE, increase in PHE tolerance, or improvement in clinical symptoms of PKU are all valid indications to continue a particular therapy.
  • Genetic counseling should be provided as an ongoing process for individuals with PKU and their families.

The complete medical and dietary guidelines are published on the National PKU Alliance website.  

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ACMG and GMDI Develop First Guidelines for Medical and Dietary Treatment of PKU
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ACMG and GMDI Develop First Guidelines for Medical and Dietary Treatment of PKU
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