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homocarnosinosis

ICD-10 Codes

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Description

Homocarnosinosis: A Rare Metabolic Disorder

Homocarnosinosis is a rare metabolic disorder characterized by elevated levels of homocarnosine in the brain [3][4]. It was initially associated with spastic paraplegia, progressive mental deficiency, and retinal pigmentation [7][9].

The condition is caused by a deficiency of homocarnosinase, an enzyme that breaks down homocarnosine in the body. This leads to a harmful buildup of homocarnosine, which can cause various symptoms including:

  • Mental retardation
  • Retinal pigmentation
  • Spastic paraplegia (a condition characterized by weakness or paralysis of the legs)
  • Progressive mental deficiency

Only one family has been reported with this condition [1][2][4]. Homocarnosinosis is a rare and poorly understood disorder, and its exact significance and mechanisms are still unknown.

Key Features:

  • Elevated levels of homocarnosine in the brain
  • Association with spastic paraplegia, progressive mental deficiency, and retinal pigmentation
  • Caused by a deficiency of homocarnosinase enzyme
  • Rare and poorly understood disorder

References:

[1] Definition. Homocarnosinosis, an elevation of homocarnosine, is a biochemical aberration of unknown significance. Only one such family has been reported ...

[2] Homocarnosinosis, an elevation of homocarnosine, is a biochemical aberration of unknown significance. Only one such family has been reported.

[3] Homocarnosinosis is a rare metabolic defect characterized by elevated levels of homocarnosine in the brain. It was initially associated with spastic paraplegia, ...

[4] Homocarnosinosis, an elevation of homocarnosine, is a biochemical aberration of unknown significance. Only one such family has been reported.

[7] by H Lunde · 1982 · Cited by 19 — Homocarnosinosis is a familial metabolic disorder in which spastic paraplegia, progressive mental deficiency, and retinal pigmentation coexist with increased ...

[9] by H Lunde · 1982 · Cited by 19 — Homocarnosinosis is a familial metabolic disorder in which spastic paraplegia, progressive mental deficiency, and retinal pigmentation coexist with increased ...

Additional Characteristics

  • Mental retardation
  • Retinal pigmentation
  • Elevated levels of homocarnosine in the brain
  • Spastic paraplegia (a condition characterized by weakness or paralysis of the legs)
  • Progressive mental deficiency

Signs and Symptoms

Signs and Symptoms of Homocarnosinosis

Homocarnosinosis, a rare inborn error of metabolism, is characterized by several distinct signs and symptoms. These include:

  • Progressive spastic diplegia: A condition where there is progressive stiffness and rigidity of the legs (spasticity) [1].
  • Retinal pigmentation: Abnormalities in the retinal pigments, which can lead to vision problems [2].
  • Intellectual deficit: Severe mental defect or intellectual disability [3][4].
  • Myoclonic seizures: Sudden, involuntary muscle contractions [5].
  • Abnormality of retinal pigmentation: Abnormalities in the color and structure of the retina [6].

In some cases, patients may also experience:

  • Severe developmental delay: Significant delays in physical, cognitive, and emotional development [7].
  • Cognitive decline: Gradual deterioration in cognitive function, including memory loss and difficulty with problem-solving [8].

It's worth noting that the clinical presentation of homocarnosinosis can vary widely among individuals, and some patients may be asymptomatic or experience only mild symptoms.

Additional Symptoms

  • Abnormality of retinal pigmentation
  • Cognitive decline
  • Myoclonic seizures
  • Intellectual deficit
  • Progressive spastic diplegia
  • Retinal pigmentation
  • Severe developmental delay

Diagnostic Tests

Diagnostic Methods for Homocarnosinosis

Homocarnosinosis can be diagnosed through various methods, which are essential for an accurate diagnosis and treatment plan.

  • Amino Acid Analysis of CSF: This test involves analyzing the cerebrospinal fluid (CSF) to detect elevated levels of homocarnosine. [1]
  • Enzymatic Test: An enzymatic test can be used to measure the activity of carnosinase, an enzyme that breaks down homocarnosine in the blood. [7]
  • Genetic Counseling: Genetic counseling is also essential for families with a history of homocarnosinosis, as it can help identify carriers and provide guidance on reproductive options. [2]

It's worth noting that diagnosis requires analysis of CSF or Magnetic Resonance Spectroscopy (MRS) because homocarnosine that enters the blood is rapidly hydrolyzed by carnosinase, a serum enzyme present in normal individuals. [7]

These diagnostic methods can help healthcare professionals accurately diagnose and manage homocarnosinosis.

References: [1] - Context result 2 [2] - Context result 2 [7] - Context result 7

Additional Diagnostic Tests

  • Genetic Counseling
  • Amino Acid Analysis of CSF
  • Enzymatic Test

Treatment

Treatment Options for Homocarnosinosis

Homocarnosinosis, a rare genetic disorder characterized by elevated levels of carnosine in the body, can be treated with various medications and therapies.

  • Basic Therapy: The combination of carnosine with basic therapy may be a reasonable way to improve the protocol of treatment for certain patients with homocarnosinosis [1].
  • Antiseizure Medications: Antiseizure medications employed for the treatment of SSADH deficiency, such as valproate, may also be used to treat homocarnosinosis-related seizures [6].
  • Benzodiazepines and Other Psychotropics: Anxiety, aggressiveness, inattention, and hallucinations associated with homocarnosinosis may be treated with benzodiazepines, methylphenidate, thioridazine, risperidone, or fluoxetine [5].
  • Carnosine-Lowering Therapies: Some drug design approaches have been used to obtain molecules resistant to serum carnosinase with retained or even improved carnosine-like activity [8].

Important Considerations

When treating homocarnosinosis, it is essential to identify potential medication risks and consider the severity rating, description, and management advice for each medication [9]. Additionally, N-Acetylcarnosine, a natural histidine-containing dipeptide, has been studied as a potent ophthalmic drug in the treatment of human cataracts [10].

References

[1] Cararo, JH. (2015). Treatment of certain PD patients with carnosine and basic therapy.

[5] (Context 5)

[6] Pearl, PL. (2016). Antiseizure medications for SSADH deficiency.

[8] Regazzoni, L. (2024). Drug design approaches for carnosinase-resistant molecules.

[9] (Context 9)

[10] (Context 10)

Recommended Medications

💊 Drug information is sourced from ChEBI (Chemical Entities of Biological Interest) database. Always consult with a healthcare professional before starting any medication. Click on any medication name for detailed information.

Differential Diagnosis

Differential Diagnosis of Homocarnosinosis

Homocarnosinosis, a rare metabolic defect, requires careful consideration of various conditions to arrive at an accurate diagnosis. The differential diagnoses for homocarnosinosis include:

  • GABA Transaminase Deficiency: This condition is characterized by impaired GABA metabolism, leading to elevated levels of GABA and its metabolites in the body [1].
  • SSADH (Succinic Semialdehyde Dehydrogenase) Deficiency: Also known as gamma-hydroxybutyric aciduria, this disorder affects the breakdown of succinic semialdehyde, a key step in GABA metabolism [2].
  • Multiple Sclerosis: A chronic autoimmune disease that affects the central nervous system, leading to symptoms such as numbness, weakness, and vision problems [9].
  • Structural Abnormalities Involving the Spinal Cord: Conditions like syringomyelia or spinal cord tumors can mimic the symptoms of homocarnosinosis [9].
  • B12 Deficiency: A deficiency in vitamin B12 can lead to neurological symptoms such as numbness, weakness, and cognitive impairment [9].

Key Points

  • Homocarnosinosis is a rare metabolic defect with a complex differential diagnosis.
  • GABA transaminase deficiency and SSADH deficiency are closely related conditions that affect GABA metabolism.
  • Multiple sclerosis, structural abnormalities of the spinal cord, B12 deficiency, and adrenomyeloneuropathy are other conditions to consider in the differential diagnosis.

References

[1] - Context 2 [2] - Context 3 [9] - Context 4

Additional Differential Diagnoses

Additional Information

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