Niemann-Pick Disease Type C (NPC) Therapeutics Pipeline to Witness Significant Growth due to Increasing Strategic Developments in the Coming Years | Top Players are Orphazyme A/S, IntraBio Ltd., CTD Holdings, Inc., Mallinckrodt Pharmaceuticals, SOM Biotec
The disease shows its first signs in mild-to-late childhood with the insidious onset of ataxia, dementia, and vertical supranuclear gaze palsy (VSGP). The occurrence of NPC can be confirmed by biochemical testing. NPC can also be diagnosed by skin test, in order to assess how the cells, move and store cholesterol. Furthermore, testing for the cells’ ability to modify cholesterol (cholesterol esterification test) has also been used for diagnostic purpose. Recently, blood-based testing for biomarkers (oxysterols, lysosphingolipids, bile acid metabolites) and molecular gene sequencing of NPC1 and NPC2 have replaced these methods.
Download the sample copy of report at: https://www.pharmaproff.com/request-sample/1255
NPC can be managed by treating symptoms including dystonia, seizures, and cataplexy. Also, preventing secondary complications, such as chest physical therapy and increased spasticity can reduce the occurrence of NPC.
According to the research, many drugs acting as a therapeutic against NPC are administered orally. It has been found that oral route of administration is easy to use, non-invasive, and ensures high level of patient satisfaction.
In the wake of strategic developments, various companies are investing in the development of therapeutic drugs for NPC, and also plan to expand their portfolio in near future. For instance, in February 2018, Oraxion Therapeutics Inc. entered into an agreement with a U.S. based biopharmaceutical company, amounting to $125 million. The agreement provides the biopharma partner the exclusive option to license its lead asset ORX-301, for the treatment of NPC and Focal Segmental Glomerulosclerosis (FSGS). However, the name of the biopharmaceutical partner was not revealed.
Get the detailed analysis at: https://www.pharmaproff.com/report/npc-therapeutics
It has been observed that regulatory bodies are granting designations to the pipeline drugs, in order to pace up the development process of these drugs. For instance, IntraBio Inc.’s IB1001 was granted Rare Pediatric Disease Designation by the USFDA in April 2018 and Orphan Drug Designation by USFDA and EMA in February 2018 and April 2017, respectively. Similarly, Orphazyme A/S’s arimoclomol has been granted Orphan Drug Designation (in January 2015), Fast-Track Designation (in June 2016), and Rare Pediatric Disease Designation (in January 2018) by the USFDA.
Make enquiry before purchase at: https://www.pharmaproff.com/request-customization/1255
Some of the key players involved in the development of NPC therapeutics are Orphazyme A/S, IntraBio Inc., CTD Holdings Inc., Mallinckrodt plc, SOM INNOVATION BIOTECH SL, Neurotrope Inc., Actelion Pharmaceuticals Ltd., Okklo Life Sciences BV, Amicus Therapeutics Inc., Oraxion Therapeutics Inc., and COI Pharmaceuticals Inc.
Toll-free: +1-888-778-7886 (USA/Canada)
Pharma Proff, is a frontline market intelligence and consulting solutions provider catering to the information and data needs of burgeoning sectors across the world, including pharmaceutical and healthcare. With meticulously curated research reports on the industry landscape, we empower companies in the healthcare and pharmaceutical sectors to make informed business decisions and base their marketing strategies with astuteness.
This release was published on openPR.
Permanent link to this press release:
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.
You can edit or delete your press release Niemann-Pick Disease Type C (NPC) Therapeutics Pipeline to Witness Significant Growth due to Increasing Strategic Developments in the Coming Years | Top Players are Orphazyme A/S, IntraBio Ltd., CTD Holdings, Inc., Mallinckrodt Pharmaceuticals, SOM Biotec here
News-ID: 1812661 • Views: 712
More Releases from Pharma Proff
Interleukin-6 (IL-6) Inhibitors Analysis, Clinical Trails and Developments
Interleukin-6 (IL-6) inhibitors currently exhibit a proliferating pipeline with 25+ therapeutic candidates. IL-6 is a cytokine encoded by IL-6 gene, which helps in the maturation of B cells and is actively involved in the inflammatory response. The IL-6 protein is primarily produced at the sites of acute and chronic inflammation, where it is secreted into the serum and brings a transcriptional inflammatory response through IL-6? receptor. This protein is assumed to
Retinitis Pigmentosa Therapeutics Analysis, Clinical Trials and Other Developmen …
Retinitis pigmentosa therapeutics currently exhibits a proliferating pipeline with 25+ therapeutic candidates. Retinitis pigmentosa (RP) is a group of hereditary disorders that lead to the breakdown and loss of cells in the retina. Mutation in genes encoding the proteins required by retinal cells for their proper functioning, results in occurrence of RP. The disease results in partial vision loss during the earlier stage of the disease, which may lead to complete
Probiotics Therapeutics -Pipeline Analysis 2018, Clinical Trials & Results, Pate …
Probiotics are live microorganisms that produces beneficial effects in human gut. Probiotics can be taken as a dietary supplement. They prevent diarrhoea and help to reduce the symptoms of irritable bowel syndrome. Download the sample report at: https://www.pharmaproff.com/request-sample/1189 Probiotics comprise of a variety of microorganisms belonging to the Lactobacillus and Bifidobacterium groups. The only yeast probiotic that has proven to be effective is Saccharomyces boulardii. According to researchers, probiotics are effective in
Hepatorenal Syndrome Therapeutics Pipeline Analysis 2018, Clinical Trials & Resu …
Hepatorenal syndrome (HRS) is a reversible functional renal impairment, that occurs in patients with advanced liver cirrhosis or those with fulminant hepatic failure. It is characterized by a marked reduction in glomerular filtration rate and renal plasma flow, in the absence of other causes of renal failure. Download the sample report at: https://www.pharmaproff.com/request-sample/1030 The symptoms of hepatorenal syndrome are fatigue, abdominal pain, malaise, ascites, jaundice, splenomegaly, and hepatomegaly. There are two
More Releases for NPC
Methanol Market Business Growth Statistics and Key Players Insights: Methanax, S …
The report Global Methanol Market 2018 focuses on fundamental know-how of Methanol segment. The report provides comprehensive study and vivid description of drivers, limitations, openings, request factors, market size, conjectures, and patterns in the Worldwide Heading market over the time of 2018 to 2025. Besides, the report is aggregate introduction of essential and optional research discoveries. The Global Methanol Market renders deep perception of the key regional market status of
Copper Foil Market: CCP, NPC, Co-Tech, LYCT, Furukawa Electric
Qyresearchreports include new market research report “Global Copper Foil Market Research Report 2018” to its huge collection of research reports. In this report, the global Copper Foil market is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2025, growing at a CAGR of XX% between 2017 and 2025. Global Copper Foil market competition by top manufacturers, with production, price, revenue (value)
Microfiber Synthetic Leathers Market: Top Players - NPC, Duksung, Tongda Island
MarketResearchReports.Biz adds “Global Microfiber Synthetic Leathers Market Share, Size, Trends and Forecast Market Research Report” reports to its database. This report provides a strategic analysis of the Microfiber Synthetic Leathers and the growth estimates for the forecasted period. This report studies the Microfiber Synthetic Leathers market status and outlook of global and United States, from angles of players, regions, product types and end industries; this report analyzes the top players in
Global Copper Foil Market 2018 - Fukuda, CCP, KINWA, NPC, LYCT
Apex Market Reports, recently published a detailed market research study focused on the "Copper Foil Market" across the global, regional and country level. The report provides 360° analysis of "Copper Foil Market" from view of manufacturers, regions, product types and end industries. The research report analyses and provides the historical data along with current performance of the global Copper Foil industry, and estimates the future trend of Copper Foil market
Global PE Pipe Resin Market 2017 New Features & Applications – Borealis, Ineos …
Qyresearchreports include new market research report Global PE Pipe Resin Market Professional Survey Report 2017 to its huge collection of research reports. The global PE Pipe Resin market is presented here with the intention of providing a sound understanding of the current and future trends and more aspects such as those pertaining to business opportunities. Using a perfect combination of primary and secondary research, the authors of the report offer a
Copper Foil for Wireless charging Market 2017 - LS Mtron, Iljin Materials, CCP, …
Apex Research, recently published a detailed market research study focused on the "Copper Foil for Wireless charging Market" across the global, regional and country level. The report provides 360° analysis of "Copper Foil for Wireless charging Market" from view of manufacturers, regions, product types and end industries. The research report analyses and provides the historical data along with current performance of the global Copper Foil for Wireless charging industry,