{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
Search results for "ORPHAN DRUG|Designated|Treatment of Amyotrophic lateral sclerosis" in comments (approximate match)
Status:
Possibly Marketed Outside US
Source:
NCT03394950: Phase 4 Interventional Completed Stroke, Ischemic
(2018)
Source URL:
First approved in 2009
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (RACEMIC)
3-N-Butylphthalide (NBP), a family comprised of optical isomers l-3-N-butylphthalide (l-NBP) and d-3-N-butylphthalide (d-NBP), with l-NBP being an extract from seeds of Apium graveolens Linn. (celery) and dl-3-N-butylphthalide (dl-NBP), a synthetized version, has been studied for its significant neuroprotective effects. NBP showed neuroprotective effects by decreasing oxidative damage, inhibiting inflammatory responses, improving mitochondrial function, and reducing
neuronal apoptosis. NBP received approval by the State Food and Drug Administration of China for clinical use in stroke patients in 2002. It demonstrates a potential for the treatment of central nervous system diseases, including Parkinson’s disease, Alzheimer’s disease.
Status:
Possibly Marketed Outside US
Source:
NCT02356107: Phase 4 Interventional Completed Major Depressive Disorder
(2015)
Source URL:
First approved in 2004
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Creatine is an amino acid that occurs in vertebrate tissues and in urine. It is synthesized mainly in the liver from three different amino acids: arginine, glycine, and methionine. In skeletal muscles, which store 95% of it, creatine generally occurs as phosphocreatine. The rest is stored in the brain, heart, and testes. Creatine functions as part of the cell's energy shuttle. It is excreted as creatinine in the urine.
Status:
Possibly Marketed Outside US
First approved in 2002
Source:
NDA207202
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Gold chloride (AUCl3), dihydrate, also called gold trichloride or auric chlorise, is a compound comprised of gold and chloride. It exists as a chloride-bridged dimer, both as a solid and as a vapor. AUCl3 is very hygroscopic and highly soluble in water and ethanol. It is used in organic chemistry as a mild acid catalyst and as a alternative to mercury salts. It is known to cause allergic reactions in subjects with known gold allergy.
Status:
Possibly Marketed Outside US
First approved in 2000
Source:
21 CFR 348
Source URL:
Class (Stereo):
CHEMICAL (RACEMIC)
Targets:
Conditions:
Borneol, a monoterpenoid alcohol, is a component of many essential oils. Barneol occurs in nature as a single enantiomer (d- or l-, depending on the oil type) or, less frequently, as the racemate. Several studies have proved the effectiveness of borneol. In Chinese medicines borneol has been used in relieving symptoms of anxiety, fatigue and insomnia; inducing anesthesia and analgesia to alleviate abdominal pain, wounds and burns; relieving rheumatic pain, hemorrhoids, skin diseases and ulcerations of the mouth, ears, eyes or nose; to treat sore throats and skin infections, and is mainly used to treat cardiovascular and cerebrovascular diseases. Borneol has a significant therapeutic effect on neuralgia. This compound is considered a GRAS approved by the FDA as food flavor. Additionally, borneol is a fragrance ingredient. GABAA, TRPV3, TRPM8 and TRPA1 have been identified as the molecular targets of borneol.
Status:
Possibly Marketed Outside US
Source:
21 CFR 352
(2018)
Source URL:
First approved in 1987
Source:
ANDA216334
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Status:
Possibly Marketed Outside US
Source:
NCT04704518: Phase 4 Interventional Completed Photorefractive Keratectomy
(2022)
Source URL:
First approved in 1958
Source:
NDA210274
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Trehalose, a naturally occurring disaccharide of glucose that appears to function in an anhydrobiotic capacity in many organisms. Bioblast Pharma study trehalose in Phase 2 for treating patients with Oculopharyngeal Muscular Dystrophy (OPMD) and spinocerebellar ataxia, type 3. In OPMD trehalose prevents the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death. Trehalose induces autophagy via mTOR independent pathway. It activates TFEB, a master controller of lysosomal biogenesis and autophagy, by inhibiting AKT which is a negative regulator of TFEB that acts by direct phosphorylation (and inhibition) of TFEB. In addition, trehalose protects cells from hypoxic and anoxic injury and suppresses protein aggregation. In vivo studies with trehalose show cellular and behavioral beneficial effects in animal models of neurodegenerative diseases. Trehalose was in phase III clinical trial to study if it was possible to use the drug as add-on therapy in Bipolar Depression. Also in combination with hyaluronate, it can be used to treat dry eye syndrome. Trehalose protects the epithelial cells on the ocular surface, improving their resistance to the daily stresses of dry environments and tear film changes in a dry eye.
Status:
Possibly Marketed Outside US
Source:
ERIL by Asahi Kasei
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Fasudil is a potent Rho kinase inhibitor, which was developed by Asahi Kasei. The drug is used in Asia for the prevention of cerebral vasospasm in patients with subarachnoid hemorrhage.
Status:
Possibly Marketed Outside US
Source:
NCT03151707: Phase 4 Interventional Terminated Healthy
(2017)
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Status:
Possibly Marketed Outside US
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Ibudilast (KETAS®) is a non-selective cyclic nucleotide phosphodiesterase (PDE) inhibitor. It is an antithrombotic, antiasthmatic drug that is used for improving prognosis and relieving symptoms in patients suffering from ischemic stroke and for the treatment of bronchial asthma. A definitive mechanism of its action is yet to be established. However, inhibition of the release of inflammatory cytokines, inhibition of leukocyte activation, and inhibition of the expression of cell adhesion molecules have been proposed as likely mechanisms of action of ibudilast (KETAS®). It is currently in development in the US (for instance as a potential therapy for multiple sclerosis), but is approved for use in Japan.
Status:
Investigational
Source:
NCT03486990: Phase 2 Human clinical trial Completed N/A
Source URL:
Class:
MIXTURE