Excruciating Technological Advancements To Drive The Acquired Hemolytic Anemia Market

Adrenoleukodystrophy give rise to three major disease categories such as childhood cerebral form (observed between 4 to 8 years of age), adrenomyelopathy and impaired adrenal gland function (also known as Addison disease).

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The product pipeline of adrenoleukodystrophy undergoing phase III trials is as follows:

• RPI-78M
• Lenti-D
• Campath-1H + Clofarabine + Melphalan + Hematopoietic Stem Cell Transplantation
• Campath-1H + Cyclophosphamide + Busulfan + Allogeneic Stem Cell Transplantation
• Busulfan + Cyclophosphamide + Antithymocyte Globulin + Hematopoietic Stem Cell Transplantation

The major symptoms observed in a childhood cerebral form adrenoleukodystrophy patient are muscle spasms, crossed eyes (strabismus), hearing loss, seizures and other disorders related with the nervous system. In adrenomyelopathy the patients are observed with difficulty in controlling urination, muscle weakness or leg stiffness, difficulties in thinking speed and lack of visual memory.

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The adrenoleukodystrophy is diagnosed primarily with plasma very long chain fatty acid (VLCFA) examination by application of gas chromatography and/or mass spectrometery. The other diagnostics methods include chromosome studies that are carried out to understand the mutation in ABCD 1 gene and magnetic resonance imaging (MRI) scan of head.]

Adrenoleukodystrophy is treated with dietary therapy, transplant, adrenal insufficiency and gene therapy. The dietary therapy consists of prohibiting the patient for the intake of very-long chain fatty acids (VLCFA) and this is a supportive therapy to normalize the disease conditions of the patient.

The transplants are performed with allogeneic hematopoietic stem cells that assist in the demyelination process where myelin sheath is restored and its deterioration is inhibited. In gene therapy appropriate vectors are selected and modified according to the normal ABCD 1 and later these are transplanted into patient’s bone marrow or stem cell transplant.

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North America was observed to be the leading geography followed by Europe due to high prevalence rate, increasing social awareness and key players based in the same geography. Asia-Pacific and Rest of the World lack due to unavailability and inaccessibility of the diagnostic techniques, counseling bodies and modern treatments.

The key players involved in the adrenoleukodystrophy therapeutics market are Nutra Pharma Corporation and GENETIX Pharmaceuticals, Inc. Adrenoleukodystrophy is a rare disease hence the companies involved in therapeutics market of disease are few in number.