![]() ![]() We enrolled boys with cerebral adrenoleukodystrophy in a single-group, open-label, phase 2–3 safety and efficacy study. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. The most trusted, influential source of new medical knowledge and clinical best practices in the world. Information and tools for librarians about site license offerings. Valuable tools for building a rewarding career in health care. The authorized source of trusted medical research and education for the Chinese-language medical community. The most advanced way to teach, practice, and assess clinical reasoning skills. Information, resources, and support needed to approach rotations - and life as a resident. The most effective and engaging way for clinicians to learn, improve their practice, and prepare for board exams. NEW! Peer-reviewed journal featuring in-depth articles to accelerate the transformation of health care delivery.Ĭoncise summaries and expert physician commentary that busy clinicians need to enhance patient care. X linked adrenoleukodystrophy trial#NEW! A digital journal for innovative original research and fresh, bold ideas in clinical trial design and clinical decision-making. ![]()
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