In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the
missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1 …

One of the latest approved therapies for spinal muscular atrophy (SMA) is onasemnogene
abeparvovec, which transduces motor neurons with the survival of motor neuron gene. The …

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder leading to
paralysis, muscle atrophy, and death. Significant advances in antisense oligonucleotide …

The Central Nervous System (CNS) is vulnerable to a range of diseases, including
neurodegenerative and oncological conditions, which present significant treatment …

Complement-mediated thrombotic microangiopathy (TMA) in the form of atypical hemolytic
uremic syndrome (aHUS) has emerged as an immune complication of systemic adeno …

117例儿童脊髓性肌萎缩症自然病史分析- PMC Back to Top Skip to main content NIH NLM
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Background Previous studies mainly focused on risk factors in patients with mild cognitive
impairment (MCI) or dementia. The aim of the study was to provide basis for preventing MCI …

Objectives To study the natural history of spinal muscular atrophy (SMA) in Chongqing and
surrounding areas, China, and to provide a clinical basis for comprehensive management …

Résumé Dans certaines maladies neurologiques, monogéniques, une séquence anormale
d'ADN dans le génome est à l'origine de la cascade physiopathologique. La meilleure …