A61P3/12

TREATMENT OF HYPERAMMONEMIA IN PATIENTS WITH RENAL INSUFFICIENCY
20220040129 · 2022-02-10 ·

Disclosed herein are methods of for treating hyperammonemia in a patient having renal impairment, comprising administering carglumic acid to said patient at a reduced daily dosage.

COMPOSITIONS AND METHODS FOR DIAGNOSIS AND TREATMENT OF NEURODEGENERATIVE DISEASES
20210330734 · 2021-10-28 ·

Compositions including small molecule biochemicals, and salts and derivatives thereof, and methods for detection, treatment or prophylaxis of neurodegenerative diseases are provided, including compositions and methods for detecting and treating conditions that contribute to neurodegenerative diseases, including peripheral and central inflammation, amyloid-β plaque deposition in the brain, neuronal iron deposition and hyperglycemia.

Pharmaceutical composition and administrations thereof

The present invention relates to pharmaceutical compositions containing a solid dispersion of N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide including formulations of the solid dispersions into powders, granules and mini-tablets, methods for manufacturing and processing the powders, granules and mini-tablets, and methods for treating cystic fibrosis employing the pharmaceutical composition.

Therapeutic compounds and methods of use thereof

The invention provides compounds having the general Formula (I); ##STR00001##
and pharmaceutically acceptable salts thereof; wherein the variables R.sup.A, R.sup.AA, subscript n, subscript q, ring A, X.sup.2, L, subscript m, X.sup.1, R.sup.1, R.sup.2, R.sup.3, R.sup.4, R.sup.5, D and E have the meaning as described herein, and compositions containing such compounds and methods for using such compounds and compositions.

Inhibitors of NHE-mediated antiport
11147884 · 2021-10-19 · ·

The present disclosure is directed to compounds (I′) and to their use in methods for the treatment of disorders associated with fluid retention or salt overload, such as heart failure (in particular, congestive heart failure), chronic kidney disease, end-stage renal disease, liver disease, and peroxisome proliferator-activated receptor (PPAR) gamma agonist-induced fluid retention. The present disclosure is also directed to compounds (I′) and their use in methods for the treatment of hypertension. The present disclosure is also directed to compounds (I′) and to their use in methods for the treatment of gastrointestinal tract disorders, including the treatment or reduction of pain associated with gastrointestinal tract disorders. ##STR00001##

Inhibitors of NHE-mediated antiport
11147884 · 2021-10-19 · ·

The present disclosure is directed to compounds (I′) and to their use in methods for the treatment of disorders associated with fluid retention or salt overload, such as heart failure (in particular, congestive heart failure), chronic kidney disease, end-stage renal disease, liver disease, and peroxisome proliferator-activated receptor (PPAR) gamma agonist-induced fluid retention. The present disclosure is also directed to compounds (I′) and their use in methods for the treatment of hypertension. The present disclosure is also directed to compounds (I′) and to their use in methods for the treatment of gastrointestinal tract disorders, including the treatment or reduction of pain associated with gastrointestinal tract disorders. ##STR00001##

FGFR INHIBITOR FOR USE IN THE TREATMENT OF HYPOPHOSPHATEMIC DISORDERS

The present invention relates generally to 3-(2,6-Dichloro-3,5-dimethoxy-phenyl)-1-{6-[4-(4-ethyl-piperazin-1-yl)-phenylamino]-pyrimid-4-yl}-1-methyl-urea or a pharmaceutically acceptable salt or solvate thereof or a pharmaceutical composition comprising 3-(2,6-Dichloro-3,5-dimethoxy-phenyl)-1-{6-[4-(4-ethyl-piperazin-1-yl)-phenylamino]-pyrimid-4-yl}-1-methyl-urea or a pharmaceutically acceptable salt or solvate thereof for use in the treatment of X-linked hypophosphatemic rickets (XLH), autosomal dominant hypophosphatemic rickets (ADHR), autosomal recessive hypophosphatemic rickets (ARHR), tumor-induced osteomalacia, post-renal transplant hypophosphatemia, epidermal nevus syndrome, osteoglophonic dysplasia or McCune-Albright syndrome.

PHARMACEUTICAL-GRADE FERRIC ORGANIC COMPOUNDS, USES THEREOF AND METHODS OF MAKING SAME

The present invention discloses pharmaceutical-grade ferric organic compounds, including ferric citrate, which are soluble over a wider range of pH, and which have a large active surface area. A manufacturing and quality control process for making a pharmaceutical-grade ferric citrate that consistently complies with the established Manufacture Release Specification is also disclosed. The pharmaceutical-grade ferric organic compounds are suitable for treating disorders characterized by elevated serum phosphate levels.

COMPOSITIONS AND METHODS FOR THE SUSTAINED RELEASE OF BETA-ALANINE
20210260005 · 2021-08-26 ·

Methods and compositions are provided for increasing the anaerobic working capacity of muscle and other tissues. Also provided are compositions formulated for the sustained release of free beta-alanine. Also provided is a dietary supplement formulated, for example, as a solid food product, an edible suspension, liquid or semi-liquid as described herein.

HETEROAROMATIC COMPOUNDS AND THEIR USE AS DOPAMINE D1 LIGANDS

The present invention provides, in part, compounds of Formula I:

##STR00001##

and pharmaceutically acceptable salts thereof; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds or salts, and their uses for treating D1-mediated (or D1-associated) disorders including, e.g., schizophrenia (e.g., its cognitive and negative symptoms), cognitive impairment (e.g., cognitive impairment associated with schizophrenia, AD, PD, or pharmacotherapy therapy), ADHD, impulsivity, compulsive gambling, overeating, autism spectrum disorder, MCI, age-related cognitive decline, dementia, RLS, Parkinson's disease, Huntington's chorea, anxiety, depression, MDD, TRD, bipolar disorder, chronic apathy, anhedonia, chronic fatigue, post-traumatic stress disorder, seasonal affective disorder, social anxiety disorder, post-partum depression, serotonin syndrome, substance abuse and drug dependence, drug abuse relapse, Tourette's syndrome, tardive dyskinesia, drowsiness, excessive daytime sleepiness, cachexia, inattention, sexual dysfunction, migraine, SLE, hyperglycemia, atherosclerosis, dislipidemia, obesity, diabetes, sepsis, post-ischemic tubular necrosis, renal failure, hyponatremia, resistant edema, narcolepsy, hypertension, congestive heart failure, postoperative ocular hypotonia, sleep disorders, and pain.