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an effective treatment would be expected to augment striatal synaptic connectivity, protect SN neurons from underlying death inducers, and ideally stimulate the replacement of lost SN neurons from preexisting pools of neural stem cells

April 29 Product: X4 Pharmaceuticals Xolremdi Indication: WHIM Syndrome The FDA approved X4 Pharmaceuticals Xolremdi Monday as the first targeted treatment for WHIM syndrome, an ultra-rare immunodeficiency disease named for its four characteristics: warts, hypogammaglobulinemia, infections and myelokathexis

PMID: 38856181)

Some patients benefit from splitting the dose into two injections per week (e.g., Monday/Thursday) to reduce the peak-to-trough variation