Abstract: The disclosure provides methods of preventing or treating DMD in a mammalian subject, reducing risk factors associated with DMD, and/or reducing the likelihood or severity of DMD. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide.

Abstract: The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms, such as exercise-induced acid acidosis, and increases in plasma levels of growth factor 15 (GDF-15) and/or fibroblast growth factor (FGF-21) in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom.

Abstract: The disclosure provides methods of preventing or treating Barth Syndrome in a mammalian subject, reducing risk factors associated with Barth Syndrome, and/or reducing the likelihood or severity of Barth Syndrome. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to increase expression of TAZ1 in subjects in need thereof.

Abstract: The disclosure provides methods of preventing, ameliorating or treating disruption of mitochondrial function and symptoms thereof. The methods provide administering aromatic-cationic peptides in effective amounts to prevent, treat or ameliorate the disruption of mitochondrial oxidative phosphorylation in a cell such as that found in a subject suffering from, or predisposed to a mitochondrial disease or disorder. In some embodiments, the methods comprise administering to a subject suffering from, or at risk for a mitochondrial disease or disorder, an effective amount of an aromatic-cationic peptide to subjects in need thereof.

Abstract: The present disclosure provides novel methods for treating, preventing, ameliorating, inhibiting and/or delaying the onset of tauopathies. The methods comprise administering to the subject an effective amount of a small molecule peptidomimetic, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

Abstract: The present disclosure provides methods for treating, preventing, ameliorates, inhibits, or delaying the onset of Leigh syndrome as well as methods for ameliorating, inhibiting, ameliorates, inhibits, or delaying the onset of its associated signs and/or symptoms in a subject in need thereof comprising administering to the subject various compounds, mixtures of compounds or compositions, formulations or medicaments derived therefrom. The present disclosure further provides compositions, formulations or medicaments and related uses for the treatment, prevention ameliorating, inhibiting, or delaying the onset of Leigh syndrome in a subject and addressing its associated signs and/or symptoms.

Abstract: The present technology provides methods and compositions for treating, preventing and/or delaying (slowing) the progression of age-related macular degeneration (AMD). In particular, the present technology relates to the use of elamipretide in effective amounts to treat, prevent and/or delay (slow) the progression of photoreceptor loss, AMD, GA secondary to AMD, non-exudative (dry) age-related macular degeneration, intermediate age-related macular degeneration, age-related macular degeneration with photoreceptor loss or non-exudative (dry) age-related macular degeneration with photoreceptor loss in mammalian subjects.

Abstract: Disclosed are analogs of elamipretide (MTP-131). The compounds are useful for the treatment and prevention of ischemia-reperfusion injury (e.g., cardiac ischemia-reperfusion injury) or myocardial infarction.

Abstract: Disclosed are various crystalline salt forms of D-Arg-Dmt-Lys-Phe-NH2.

Abstract: The disclosure provides methods for treating muscular dystrophy (MD), such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) in a mammalian subject. The methods comprise administering to the subject an effective amount of a peptide such as H-D-Arg-2,6-Dmt-Lys-PHe-NH2 (SEQ ID NO: 2) (a.k.a. MTP-131 or elamipretide), or a pharmaceutically acceptable salt, hydrate, solvate and/or tautomer thereof, in combination with at least one phosphorodiamidate morpholino oligomer (PMO) or peptide-conjugated PMO (PPMO). The methods are particularly useful for increasing dystrophin expression levels in subjects diagnosed with, and/or being treated for, MD in an amount greater than what would be possible by treatment with the PMO or PPMO alone.

Abstract: The present disclosure provides methods for treating, preventing, ameliorating, inhibiting or delaying the onset of injection site reactions associated with the subcutaneous administration of bevemipretide, or a pharmaceutically acceptable salt thereof. The methods may involve administration of inhibitors of the MRGPRX2 receptor and/or inhibitors of mast cell degranulation. In some cases, the methods involve administration of an effective amount of a flavonoid, a coumarin, a phenol, a terpenoid, mometasone furoate, tacrolimus, quercetin, diphenhydramine and/or ice.

Abstract: The disclosure provides compositions and methods relating to aromatic-cationic peptides. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof. For example, the peptides may be administered to subjects in need of a mitochondrial-targeted antioxidant.

Abstract: The present disclosure provides a combination therapy for treating, preventing, inhibiting, ameliorating, or delaying the onset of Friedreich's ataxia in a subject in need thereof comprising administering to the subject both elamipretide and omaveloxolone or pharmaceutically acceptable salts, stereoisomers, mixtures of stereoisomers, tautomers, hydrates, and/or solvates thereof. The present disclosure further provides an embodiment of a combination product suitable for administration to said subject.

Abstract: The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing ?-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondria-targeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof.

Abstract: The disclosure provides compositions and methods relating to aromatic-cationic peptides. The methods comprise administering to the subject an effective amount of an aromatic-cationic peptide to subjects in need thereof. For example, the peptides may be administered to subjects in need of a mitochondrial-targeted antioxidant.

Abstract: The present technology provides methods of generating the peptides, and pharmaceutically acceptable salts of the peptides and intermediates thereof. In some embodiments, the peptide is D-Arg-2?6?-Dmt-Lys-Phe-NH2.

Abstract: The disclosure provides therapeutic compounds, compositions (e.g., therapeutic agents or medicaments) and methods for preventing or treating mitochondrial disease such as Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with mitochondrial disease, such as Friedreich's ataxia, and/or reducing the likelihood or severity of mitochondrial disease such as Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions. In some instances, the intermediates may themselves by therapeutic agents or prodrugs of therapeutic agents (e.g. reduced forms of the therapeutic compounds).

Abstract: The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).

Abstract: The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).

Abstract: The disclosure generally describes methods of preventing or treating dominant optic atrophy. The methods comprise administering an effective amount of an aromatic-cationic peptide to subjects in need thereof. The present technology relates generally to the treatment or prevention of Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA) in mammals through administration of therapeutically effective amounts of aromatic-cationic peptides to subjects in need thereof. In one aspect, the present disclosure provides a method of treating or preventing dominant optic atrophy in a mammalian subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of a peptide.