The present invention describes methods for treating idiopathic short stature. Described herein are also methods of increasing a subject’s height. The methods involve administering an effective amount of a pan FGFR inhibitor or a selective FGFR inhibitor to the subject. An example of an FGRF inhibitor used in the methods described herein is erdafitinib.

The present invention relates to conjugate compounds which comprise a peptide moiety (a) which is preferably a hydrophobic modified preS-derived peptide of hepatitis B virus or a respective cyclic peptide, and a NTCP substrate moiety (b), which is preferably a bile acid. The present invention further relates to pharmaceutical compositions comprising at least one conjugate compound. The present invention further relates to medical uses of said conjugate compounds and the pharmaceutical compositions, such as in the diagnosis, prevention and/or treatment of a liver disease or condition, and/or in the inhibition of HBV and/or HDV infection. The present invention further relates to methods of diagnosis, prevention and/or treatment of a said diseases and/or infections.

Compositions that comprise salts, compounds and complexes of .sup.64Zn-enriched zinc, such as .sup.64Zn-enriched zinc aspartate, and further optionally include .sup.85Rb-enriched rubidium salt compounds of general formula 1, below, wherein each of R.sub.1 through R.sub.14 is independently selected from H, OH, F, Cl, Br, I, C.sub.1-C.sub.6 alkyl, C.sub.1-C.sub.6 alkoxy, and NO.sub.2, such as the compound of Formula 1 in which R.sub.3 is CH.sub.3 and all other R groups are H for use to treat a neurodegenerative disease (NDD), such as Parkinson's disease (PD). Methods that entail administering such compositions to treat an NDD, such as PD, optionally in combination with any other treatment for an NDD such as for PD.

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In an embodiment of the invention, a composition for treating a cell population comprises a medicant. The medicant moiety can be an illudofulvene analog. In an embodiment of the invention, a composition for treating a cell population comprises an Affinity Medicant Conjugate (AMC). The affinity moiety can be an antibody, an antibody fragment, a receptor protein, a peptidic growth factor, an anti-angiogenic protein, a specific binding peptide, protease cleavable peptide, a glycopeptide, a peptide, a peptidic toxin, a protein toxin and an oligonucleotide. The affinity moiety can be covalently bound to the medicant via a linker.

In an embodiment of the invention, a composition for treating a cell population comprises a medicant. The medicant moiety can be an illudofulvene analog. In an embodiment of the invention, a composition for treating a cell population comprises an Affinity Medicant Conjugate (AMC). The affinity moiety can be an antibody, an antibody fragment, a receptor protein, a peptidic growth factor, an anti-angiogenic protein, a specific binding peptide, protease cleavable peptide, a glycopeptide, a peptide, a peptidic toxin, a protein toxin and an oligonucleotide. The affinity moiety can be covalently bound to the medicant via a linker.

Disclosed are spiro-lactam compounds, and pharmaceutically acceptable salts thereof, that can ameliorate or treat a viral infection in a subject in need thereof. The disclosure also includes conjugates of such compounds of viral protease inhibitors with the cysteine at position or an equivalent active site cysteine on the coronavirus main protease (Mpro).

The present invention relates to a mixed-charge nanoparticle for inducing selective death of cancer cells and a use thereof. The mixed-charge nanoparticle of the present invention is localized and crystallized specifically in cancer cell lysosomes through a pH-dependent aggregation behavior due to the balance between positively charged ligands and negatively charged ligands on the surface thereof and can induce lysosomal membrane permeabilization (LMP) and lysosomal cell death mediated thereby, like cationic amphiphilic drugs (CADs) Exhibiting a cancer cell-specific death effect, the nanoparticles of the present invention can surmount the limited medical use of conventional cationic nanoparticles due to the non-specific cytotoxicity thereof. Particularly, the nanoparticles of the present invention do not exhibit toxicity to the human body and normal cells, thus finding useful applications in medical and medicinal uses such as for prevention and treatment of solid cancer, blood cancer, and tumors.

The present invention relates to a mixed-charge nanoparticle for inducing selective death of cancer cells and a use thereof. The mixed-charge nanoparticle of the present invention is localized and crystallized specifically in cancer cell lysosomes through a pH-dependent aggregation behavior due to the balance between positively charged ligands and negatively charged ligands on the surface thereof and can induce lysosomal membrane permeabilization (LMP) and lysosomal cell death mediated thereby, like cationic amphiphilic drugs (CADs) Exhibiting a cancer cell-specific death effect, the nanoparticles of the present invention can surmount the limited medical use of conventional cationic nanoparticles due to the non-specific cytotoxicity thereof. Particularly, the nanoparticles of the present invention do not exhibit toxicity to the human body and normal cells, thus finding useful applications in medical and medicinal uses such as for prevention and treatment of solid cancer, blood cancer, and tumors.

The present invention relates to a targeted protease degradation (TED) platform, and specifically to a conjugate of target molecule-linker-E3 ligase ligand as shown in formula I, R.sub.T-L1-R.sub.E3 (formula I), wherein R.sub.T is a monovalent group of the target molecule, R.sub.E3 is a monovalent group of the E3 ligase ligand, L1 is the linker linking A and B, and L1 is as shown in formula II below: —W-L2-W.sup.2— (II).

Provided are compositions and methods useful in regenerating damaged tissue, especially cardiac tissue, by delivering to the site of injury an miRNA that can reduce, for example, the expression of phosphatase and tensin homolog (PTEN). The compositions and methods of the disclosure may be generally applied to deliver an miRNA to a cell or tissue such as, but not limited to, a neuron, a smooth muscle cell, or a tumor cell. The compositions comprise a transmembrane carrier peptide conjugated, optionally by a linker, to an oligonucleotide complementary to an miRNA. The carrier peptide facilitates the entry of the miRNA into cells and for delivery to a tissue of an animal or human may be mixed with an extracellular matrix-derived hydrogel carrier.