Use of myostatin inhibitors and combination therapies
AssigneeScholar Rock, Inc.
InventorsKimberly Long, Adriana Donovan, Yung Chyung, Michelle Straub
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Abstract
The present disclosure relates to the treatment of muscle conditions, such as SMA, with the use of an agent that inhibits myostatin signaling. The disclosure includes combination therapies that include a myostatin inhibitor and a neuronal corrector.
Claims22
A method of treating a neuromuscular disease in a subject, the method comprising administering to the subject a myostatin inhibitor and a neuronal corrector therapy in amounts effective to treat the neuromuscular disease,
- wherein:
- i) the neuromuscular disease affects a target muscle that is in an anabolic state;
- ii) the subject has impaired neurological signaling between a motor neuron and a target muscle, wherein the target muscle has retained or regained at least partial functional innervation by the motor neuron;
- and
- iii) the neuromuscular disease affects a target muscle that is enriched with type II fibers;
- wherein the subject receives the myostatin inhibitor and the neuronal corrector therapy within six months of one another;
- wherein the neuronal corrector therapy comprises an agent capable of correcting a neuronal defect in the motor neuron;
- and
- wherein the neuromuscular disease is spinal muscular atrophy (SMA).
A method of treating spinal muscular atrophy (SMA) in a subject, the method comprising:
- (i) selecting a subject, wherein the subject has SMA and is a pediatric subject or a young adult who is still growing and anabolically active;
- and
- (ii) administering to the subject a selective myostatin inhibitor and an SMN corrector therapy in amounts effective to treat SMA.
A method of treating spinal muscular atrophy (SMA) in a subject, the method comprising:
- (i) selecting a subject, wherein the subject:
- (a) has SMA and is a pediatric subject or a young adult who is still growing and anabolically active;
- and (b) is on an SMN corrector therapy;
- and
- (ii) administering to the subject a selective myostatin inhibitor in an amount effective to treat SMA.
Dependencies are inferred from the claim text.