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12:00 AM
Feb 13, 2012
 |  BioCentury  |  Emerging Company Profile

Halo: Treating DMD symptoms

Halo targeting Duchenne Muscular Dystrophy with patient group funding

Most companies developing therapeutics for Duchenne muscular dystrophy are targeting the genetic defects that cause the disease. Halo Therapeutics LLC, a company funded by two patient groups, thinks there is still room for treatments to improve the manifestations of DMD. Its HT-100 is designed to address the fibrosis and lack of muscle regeneration that characterize the disease.

DMD is an X chromosome-linked disease that arises from a mutation in the dystrophin gene, resulting in a lack of the dystrophin protein, which acts as a scaffold and shock absorber in muscle fibers.

DMD patients typically experience progressive muscle weakness starting in early childhood that can lead to death in early adulthood, usually due to cardiac or pulmonary dysfunction.

There are no drugs specifically approved for DMD; the current standard of care is corticosteroids, which only delay disease progression. Most efforts to develop therapeutics have been focused on fixing the defective dystrophin gene through exon skipping or up-regulating production of native utrophin.


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