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10.1128/msystems.00261-19 Msystems
Skysona treats cerebral adrenoleukodystrophy, a rare disease that allows very-long-chain fatty acids to accumulate in the brain and cause damage
Symptoms such as pelvic pain, heavy periods, or pain during intercourse may emerge in the teenage years, but its also common for symptoms to be subtle or delayed, sometimes not appearing until later in life, especially when fertility challenges arise
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