تالیف: دکتر پروانه کریم زاده
Adrenoleukodystrophy (ALD) is a rare neurodegenerative disease in males, which has two important presentations; Childhood cerebral onset, the most common form, tends to progress rapidly and typically death occurs by age 10, although lifespan can be increased with early diagnosis and early bone marrow transplant.
In late-onset, symptoms progress slowly and may not appear until adulthood. It is important that some boys with X-linked gene do not develop clinical findings. Women are the carrier and about half of the females become symptomatic and show neurological manifestation but progress slower than the adult type of ALD.
In Iran, almost all patients with neurodegenerative disorders are referred to referral Pediatric Neurology centers in Tehran for advance evaluation and diagnosis.
We have diagnosed many patients with X-linked ALD in Iran that some of them managed with Bone Marrow Transplant, and we have had a good result in these patients.
Also, in some patients with normal MRI and high Very Long-chain Fatty Acid that the diagnosis was confirmed by the genetic study (Especially in siblings of our patients and after the screening of family for ALD) we used Lorenzo Oil and they did not show any signs& symptoms of ALD for a long time follow up.
According to our experience in ALD in Iran, we held the 7th Iranian Congress of Neurometabolic diseases in Tehran/Mofid Children Hospital under the title of ALD and Lorenzo Oil.
This book is the result of this Congress, and we collected the articles of highly experienced professors in Iran and had published as Adrenoleukodystrophy book.