Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-11-30 | cannabidivarin | GW Research (UK) | Rett syndrome |
Granting of the orphan status in the US |
2016-11-09 | 6-(4-(4-(2,3-dichlorophenyl)-piperazin-1-yl)-butoxy)-2H-benzo[b][1,4]oxazin-3(4H)-one hydrochloride | Reviva Pharmaceuticals (USA - CA) | pulmonary arterial hypertension |
Granting of the orphan status in the US |
2016-09-01 | recombinant adeno-associated virus vector serotype 9 expressing human iduronate-2-sulfatase | Research Institute at Nationwide Children's Hospital Center for Gene Therapy (USA- OH) | mucopolysaccharidosis II, also called Hunter syndrome A. |
Granting of the orphan status in the US |
2016-09-01 | recombinant adeno-associated virus vector serotype 9 expressing human N-Sulfoglucosamine Sulfohydrolase | Research Institute at Nationwide Children's Hospital Center for Gene Therapy (USA - OH) | mucopolysaccharidosis type III A (Sanfilippo syndrome A) |
Granting of the orphan status in the US |
2016-09-07 | 6-fluoro-(18F)-L-3,4-dihydroxyphenylalanine | Advanced Imaging Projects (USA - Fl) | diagnostic for management of patients with Lesch-Nyhan syndrome |
Granting of a patent |
2016-05-11 | 6-methoxy-3-(3',4',5'-trimethoxybenzoyl) indole | SynCore Biotechnology (Taiwan) | cholangiocarcinoma |
Granting of the orphan status in the US |
2016-12-22 | silmitasertib | Senhwa Biosciences (Taiwan) | cholangiocarcinoma |
Granting of the orphan status in the US |
2016-08-29 | 9,10-dimethoxy-5,6-dihydro-[1,3] dioxolo[4,5-g]isoquinolino[3,2-alpha]isoquinolin-7-ium(R)-4-((3R,5S,7S,8R,9S,10S,13R,14S,17R)-3,7-dihydroxy-10,13-dimethyhexadecahydro-1H-cyclopenta[alpha] | HighTide Biopharma (Australia) | primary sclerosing cholangitis |
Granting of the orphan status in the US |
2016-07-25 | recombinant, adeno-associated virus serotype 5 vector, containing the transgene, which encodes for the human protein, frataxin | Agilis Biotherapeuties (USA - MA) | Friedreich's Ataxia |
Granting of the orphan status in the US |
2016-05-23 | 9-cis, 12-cis-11,11-d2-linoleic acid ethyl ester | Retroitope (USA - CA) | Friedreich's ataxia |
Granting of the orphan status in the US |
2016-05-18 | Copper 64-DOTA-TATE | RadioMedix (USA - TX) | diagnostic for the management of neuroendocrine tumors |
Granting of the orphan status in the US |
2016-05-03 | acyclovir | Cumulus Pharmaceuticals (USA - WY) | herpetic keratitis |
Granting of the orphan status in the US |
2016-10-14 | adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8 | Solid GT (USA - MA) | Duchenne muscular dystrophy |
Granting of the orphan status in the EU |
2016-07-13 | recombinant human biglycan | Tivorsan Pharmaceuticals (USA - RI) | Duchenne muscular dystrophy |
Granting of the orphan status in the US |
2016-09-01 | adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX) | Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences | hemophilia B | Granting of the orphan status in the US |
0000-00-00 | ||||
0000-00-00 | ||||
2017-01-03 | ||||
0000-00-00 | Compound: | vkh | vki | Company acquisition |
0000-00-00 | Compound: | Laboratory | Indication |
© 2024 Biopharmanalyses - Powered by Samacom+