Provided is a bilirubin derivative-based ultrasound contrast agent for diagnosis and treatment. The fine particles including the bilirubin derivative are sensitive to reactive oxygen species (ROS), bind with hydrophobic drugs, and can effectively chelate metals such as iron oxide nanoparticles. Therefore, the fine particle of the present invention can be used as an ultrasound contrast agent for diagnosis, as a magnetic resonance imaging contrast agent, or as a carrier for hydrophobic drugs or platinum-based drugs.

This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.

This invention relates to chemotherapeutic drug implants, and more particularly, to biodegradable chemotherapeutic drug implants comprising irinotecan, or derivatives or pharmaceutically acceptable salts thereof. The present invention also relates to processes for the preparation of these chemotherapeutic drug implants and to their use in therapy, in particular in treating brain tumours.

A composition, and method for a targeted drug delivery is disclosed in treating central nervous system injury, including blast hearing loss, traumatic brain injury (TBI) and the like, by administering a subject with nanoparticle-based minocycline formulations. The formulation contains nanoparticles encapsulating minocycline for neuroprotective effect in TBI. Albumin nanoparticle-based minocycline formulations provide enhanced delivery to brain, and reduced toxicity at minimal dosage for treating a subject suffering from central nervous system injury including blast induced traumatic brain injury (bTBI). Nanoparticle administered at minimal dose in rat blast TBI model crossed blood-brain barrier (BBB) and enhanced therapeutic concentration compared to free minocycline. Provided is an effective and safe minocycline delivery in TBI with minimal or no toxicity for neuroprotective therapy. Studies indicate performance for behavioral (acute and chronic), pathological (chronic) and hearing loss mitigation using the disclosed drug and nanoparticles in rat moderate bTBI model.

The present disclosure provides methods for treating or preventing Huntington's disease (HD) in a subject in need thereof, comprising administering a therapeutic agent that activates mTORC 1 function and/or increases Ras Homolog Enriched in Striatum (Rhes) level in the subject's brain as compared to the function or level in the subject prior to treatment, and methods for modulating mHTT-associated metabolic phenotypes and/or reversal of striatal atrophy by administering a therapeutic agent that activates mTORC1 function and/or increases Ras Homolog Enriched in Striatum (Rhes) level in the subject's brain as compared to the function or level in the subject prior to treatment.

Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

The present invention relates to compositions and methods for the delivery of agents to a subject, particularly to the central nervous system (CNS).

Disclosed herein are methods for promoting neuronal outgrowth in a subject with a neuronal lesion in their CNS by administering effective amounts of a pro-regenerative OPN fragment, optionally with IGF1 and/or BDNF. A voltage gated potassium channel blocker can also be administered. Pharmaceutical compositions, devices for administration, and kits are also disclosed.

Described herein are sustained release formulations of polymer-coated local anesthetic agents, and methods for using the same to relieve or manage pain, including postsurgical pain.

An osteogenic composition for implantation at or near a target tissue site beneath the skin is provided, the osteogenic composition comprising bone morphogenetic protein and a NEMO binding domain peptide, where the NEMO binding domain peptide reduces soft tissue inflammation at or near the target tissue site. In some embodiments, a method is provided for treating a target tissue site in a patient in need of such treatment, the method comprising implanting an osteogenic composition comprising bone morphogenetic protein and a NEMO binding domain peptide, where the NEMO binding domain peptide reduces soft tissue inflammation at or near the target tissue site.