A signaling pathway refers to a group of molecules within a cell — called signaling molecules —  that coordinate to regulate a particular biochemical process, such as cell division or hormone action. The pathway is also called a signal transduction cascade. Signaling molecules work by binding to specific proteins on the cell, called receptors, which then triggers the pathway.

For the treatment of muscular dystrophy, several small molecules are used as therapeutics. These small molecules can easily pass through the cell membrane, and directly act on these signaling pathway molecules.

Signaling pathways can be upregulated or downregulated based on the environmental changes the cell perceives in its immediate surroundings. They also can be upregulated or downregulated by the action of small molecule compounds.

What is signaling pathway upregulation?

Signaling pathway upregulation is a process by which the availability of molecules involved in the signaling pathway — such as proteins, mRNA, or even energy —  is increased in the cell.

Depending on the environmental cues, the cell can increase the availability of certain molecules to carry out specific functions. For example, the cell increases the availability of DNA, RNA, and proteins to meet the requirements for cell division.

Signaling pathway upregulators for the treatment of muscular dystrophy

There are several small molecules that can upregulate signaling pathways to promote the availability of energy, and synthesis of proteins required for proper muscle growth and function. These include puldysa and oxandrolone.

Puldysa (idebenone)

Puldysa (idebenone) is a synthetic analog of a molecule found in the mitochondria, called ubiquinone. Mitochondria are like the powerhouses of the cell. They generate the energy required for every biological process, including muscle growth and movement. Ubiquinone is a molecule found in the mitochondria that plays an important role in energy production.

By mimicking the function of ubiquinone, Puldysa can help increase energy production in the cells, as well as protect them from damage. Puldysa showed good efficacy in preventing loss of respiratory function in several clinical trials involving people with Duchenne muscular dystrophy (DMD).

Oxandrolone

Oxandrolone is a synthetic equivalent of the male hormone testosterone. It mimics the action of testosterone by binding to so-called androgen receptors in muscle cells. This, in turn, promotes the synthesis of several proteins that are required for muscle growth, metabolism, and repair.

While oxandrolone was shown to have some benefits in slowing muscle weakness in boys with DMD, it is not currently recommended for the long-term treatment of the condition.

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