Recent research in gene therapy and genome engineering is addressing a range of conditions, including spinal muscular atrophy (SMA), leukemia, lymphoma, Duchenne muscular dystrophy, hemophilia, and sickle cell disease Woolfson (2025). This work suggests a promising direction for treating SMA and other genetic disorders.
The focus on SMA within this broader context highlights the potential for innovative treatments like gene therapy to improve outcomes for SMA patients. While the specific details of these advancements are not outlined in the snippet, the inclusion of SMA alongside other serious conditions underscores the ongoing efforts to develop effective therapies for genetic diseases (Woolfson, 2025).
Clinical Trials
This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:
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