Embodiments described herein provide a solution to the measurement of various dystrophins expressed in a cell or tissue. The solution to this problem is a specific and sensitive quantitative method to measure endogenous and/or exogenous dystrophin (e.g., engineered dystrophin) expression in skeletal muscle tissue, which provides a substantial benefit to drug development and monitoring of treatment.

The present disclosure relates to customized therapy for disease. The present disclosure also relates to aptamer-based compositions and methods for identifying, modulating and monitoring drug targets in muscular disease (e.g., Duchenne muscular dystrophy).

The present disclosure provides methods for predicting a patient's response to biglycan therapy for diseases or conditions associated with an abnormal level or activity of biglycan; disorders associated with an unstable cytoplasmic membrane, for example, due to an unstable dystrophin associated protein complex (DAPC); disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation. Examples of such diseases include muscular dystrophies, such as Duchenne's Muscular Dystrophy, Becker's Muscular Dystrophy, neuromuscular disorders and neurological disorders. This application also provides combination therapeutics, such as a biglycan therapeutic and a utrophin therapeutic.

Disclosed herein are methods for diagnosing, prognosing and treating muscular dystrophy. Also disclosed are methods of determining the effectiveness of an agent for the treatment of muscular dystrophy. Provided are methods of enhancing muscle regeneration, repair, or maintenance in a subject by administering galectin, such as Galectin-1 and/or Galectin-3 to a subject in need thereof. Also disclosed are methods of increasing or maintaining muscle strength and/or bone density in a subject by administering an effective amount of a Galectin-1 composition, Galectin-3 composition or a combination thereof to the subject in need thereof. Methods of preventing, inhibiting and/or reducing muscle loss and/or bone loss in a subject by administering an effective amount of a Galectin-1 composition, Galectin-3 composition or a combination thereof to the subject in need thereof are disclosed.

Provided herein are antibodies that specifically bind to a peptide antigen. Also provided are methods of detecting, isolating or quantifying the peptide antigen. In some embodiments, the peptide antigen is released from dystrophin or microdystrophin by protease digestion. Also provided are methods for detecting or quantifying microdystrophin or dystrophin in a sample using the antibodies. In some embodiments, a method described herein is used to monitor the expression of microdystrophin in a subject that has been administered a nucleic acid based therapy.

Disclosed herein are methods for diagnosing, prognosing and treating muscular dystrophy. The disclosed methods can be used to diagnosis, prognosis or treat a subject with merosin-deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FHMD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD). Also disclosed are methods of determining the effectiveness of an agent for the treatment of muscular dystrophy. In an example, a method of diagnosing or prognosing a subject with muscular dystrophy includes detecting expression of Galectin-1 or Galectin-3 in a sample obtained from the subject at risk of having or having one or more signs or symptoms associated with muscular dystrophy, thereby diagnosing or prognosing the subject with muscular dystrophy. Also provided are methods of enhancing muscle regeneration, repair, or maintenance in a subject by administering galectin, such as Galectin-1 and/or Galectin-3 to a subject in need thereof.

Disclosed herein are methods for diagnosing, prognosing and treating muscular dystrophy. The disclosed methods can be used to diagnosis, prognosis or treat a subject with merosin-deficient congenital muscular dystrophy Type 1A (MDC1A), limb-girdle muscular dystrophy (LGMD), facioscapulohumeral (FHMD), Beckers muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD). Also disclosed are methods of determining the effectiveness of an agent for the treatment of muscular dystrophy. In an example, a method of diagnosing or prognosing a subject with muscular dystrophy includes detecting expression of Galectin-1 or Galectin-3 in a sample obtained from the subject at risk of having or having one or more signs or symptoms associated with muscular dystrophy, thereby diagnosing or prognosing the subject with muscular dystrophy. Also provided are methods of enhancing muscle regeneration, repair, or maintenance in a subject by administering galectin, such as Galectin-1 and/or Galectin-3 to a subject in need thereof.