A61K31/708

Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome

The invention relates generally to a pharmacological therapy for a human genetic diseases, specifically mitochondrial DNA depletion syndromes, and more specifically, thymidine kinase 2 (TK2) deficiency. The pharmacological therapy involves the administration of at least one deoxyribonucleoside monophosphate, or mixtures thereof. For the treatment of TK2 deficiency, the pharmacological therapy involves the administration of either deoxythymidine monophosphate (dTMP) or deoxycytidine monophosphate (dCMP), or mixtures thereof. This molecular bypass approach is applicable to other disorders of unbalanced nucleotide pools, especially those found in mitochondrial DNA depletion syndrome.

Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome

The invention relates generally to a pharmacological therapy for a human genetic diseases, specifically mitochondrial DNA depletion syndromes, and more specifically, thymidine kinase 2 (TK2) deficiency. The pharmacological therapy involves the administration of at least one deoxyribonucleoside monophosphate, or mixtures thereof. For the treatment of TK2 deficiency, the pharmacological therapy involves the administration of either deoxythymidine monophosphate (dTMP) or deoxycytidine monophosphate (dCMP), or mixtures thereof. This molecular bypass approach is applicable to other disorders of unbalanced nucleotide pools, especially those found in mitochondrial DNA depletion syndrome.

TREATMENT OF MITOCHONDRIAL DISEASES

The present invention provides a composition comprising one or more deoxyribonucleosides for use in the treatment of a mitochondrial DNA depletion and/or multiple deletions syndrome provided that the syndrome is not caused by a defect in the deoxyribonucleoside triphosphate (dNTP) metabolism. With the use of the invention there is a recovery in mitochondrial DNA levels independently from the severity of the patient's disease, which confers a great therapeutic value to the invention.

TREATMENT OF MITOCHONDRIAL DISEASES

The present invention provides a composition comprising one or more deoxyribonucleosides for use in the treatment of a mitochondrial DNA depletion and/or multiple deletions syndrome provided that the syndrome is not caused by a defect in the deoxyribonucleoside triphosphate (dNTP) metabolism. With the use of the invention there is a recovery in mitochondrial DNA levels independently from the severity of the patient's disease, which confers a great therapeutic value to the invention.

TREATMENT OF MITOCHONDRIAL DISEASES

The present invention provides a composition comprising one or more deoxyribonucleosides for use in the treatment of a mitochondrial DNA depletion and/or multiple deletions syndrome provided that the syndrome is not caused by a defect in the deoxyribonucleoside triphosphate (dNTP) metabolism. With the use of the invention there is a recovery in mitochondrial DNA levels independently from the severity of the patient's disease, which confers a great therapeutic value to the invention.

USE OF FXR AGONISTS FOR TREATING AN INFECTION BY HEPATITIS D VIRUS

The present invention relates to the use of a farnesoid X receptor (FXR) agonist for the treatment of hepatitis D infection.

USE OF FXR AGONISTS FOR TREATING AN INFECTION BY HEPATITIS D VIRUS

The present invention relates to the use of a farnesoid X receptor (FXR) agonist for the treatment of hepatitis D infection.

Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
11628182 · 2023-04-18 · ·

Deoxynucleotide prodrugs for treatment of diseases characterized by unbalanced nucleotide pools are provided herein.

Nucleotide Applications
20230158056 · 2023-05-25 ·

A nucleotide is useful in preparation of drugs for preventing and treating allergic rhinitis and other diseases or in functional foods for relieving allergic rhinitis symptoms. The nucleotide is a mixture of five nucleotides, i.e., CMP, AMP, UMP, GMP and IMP, with a small molecular weight, rapid absorption into the human body and high bioavailability. The intake of nucleotide can play a role of treating allergic diseases by alleviating allergic rhinitis symptoms, relieving allergy-induced splenomegaly, significantly reducing histamine levels in serum and nasal lavage fluid, and effectively regulating inflammatory factors.

Nucleotide Applications
20230158056 · 2023-05-25 ·

A nucleotide is useful in preparation of drugs for preventing and treating allergic rhinitis and other diseases or in functional foods for relieving allergic rhinitis symptoms. The nucleotide is a mixture of five nucleotides, i.e., CMP, AMP, UMP, GMP and IMP, with a small molecular weight, rapid absorption into the human body and high bioavailability. The intake of nucleotide can play a role of treating allergic diseases by alleviating allergic rhinitis symptoms, relieving allergy-induced splenomegaly, significantly reducing histamine levels in serum and nasal lavage fluid, and effectively regulating inflammatory factors.