Natural History of MLIV

Updated on 8 November 2020


Natural History of MLIV


Subjects with MLIV are seen annually for 5 years to assess ongoing for physical and neurological changes associated with the disease.

Condition Growth Hormone Deficiencies/Abnormalities
Clinical Study IdentifierTX137397
Last Modified on8 November 2020


Yes No Not Sure

Inclusion Criteria

Confirmed diagnosis of MLIV
Able to travel to Dallas one time every 12 months for 5 years – visit requires approximately 3 days
Able to tolerate necessary modest amount of blood drawing, performance of neuro-imaging studies including MRI of the head under anesthesia and ophthalmological examinations under monitored sedation (not required for all subjects)

Exclusion Criteria

No diagnosis of MLIV
Unable to travel to Dallas one time every 12 months for 5 years
Unable to tolerate Inclusion testing as required
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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