GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid). The investigators will examine the efficacy and safety of aceneuramic acid (SA-ER tablets) 6g daily for 48 weeks in patients with GNE myopathy in a placebo-controlled, double-blind, controlled trial.

Start Date08 Feb 2021

Sponsor / Collaborator

Nobelpharma Co., Ltd.

ChiCTR1800017192

/ SuspendedNot ApplicableIIT

Efficacy evaluation of compound oligosaccharide-sialic acid administration during pregnancy on maternal-infant gut microbiota modification

Start Date01 Aug 2019

Sponsor / Collaborator

Peking University First Hospital

NCT03775967

/ CompletedNot ApplicableIIT

Estimation of Sialic Acid and IL10 Levels in Stage 1 and 2 Periodontitis Patients

Sialic acid (SA) is the generic term given to a family of acetylated derivatives of neuraminic acid. SA is a 9 carbon monosaccharide. An important function of host SA is to regulate innate immunity. SA is present in several acute phase proteins which are known to be associated with periodontitis. There exists a need for a biomarker, for early detection of disease evolution and more robust therapy efficacy measurements. Till date, there is very little data regarding simultaneous estimation and comparison of total SA content in saliva. IL-10, an anti-inflammatory cytokine, regulates the synthesis of pro-inflammatory cytokines such as IL-1, -2, -6 and stimulates protective antibody production. IL-10 is expressed in both healthy and diseased human periodontal tissues and is reported to be reduced in patients infected with Aggregatibacter actinomycetemcomitans.
The present study was therefore undertaken with an aim, to not only assess the levels of TSA(Total sialic acid levels),but also of IL 10 levels in saliva from stage 1(mild) to stage 2(moderate) periodontitis patients

Start Date10 Oct 2018

Sponsor / Collaborator

Ajman University

100 Clinical Results associated with Aceneuramic acid

100 Translational Medicine associated with Aceneuramic acid

100 Patents (Medical) associated with Aceneuramic acid

5,206

Literatures (Medical) associated with Aceneuramic acid

01 Sep 2025·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Development and validation of a label-free HPLC-CAD method to determine total sialic acid in therapeutic proteins

Article

Author: Xu, Yuechuan Alex ; Chen, Zhi ; Zeng, Ming ; Peddicord, Michael ; Cheng, Julie ; Peters, Björn-Hendrik ; Wang, Yifei ; Qiu, Difei

Sialylation plays a crucial role in influencing the pharmacokinetic, chemical, physical, and immunogenic properties of therapeutic proteins and is commonly tracked through total sialic acid analysis. In this study, we developed a novel label-free HPLC method to monitor total sialic acid levels in protein drug products. The method utilizes a replaceable, pre-column C18 cartridge that removes protein in-line, preventing protein interference with sialic acid and contamination of the analytical column and enabling direct sample injection. The optimized mixed-mode hydrophilic interaction-ion exchange liquid chromatography (HILIC-IEX) separation condition includes a volatile mobile phase for charged aerosol detection (CAD) of sialic acids. The method was validated to demonstrate satisfactory specificity, linearity (R > 0.999), precision (RSD between 0.4 % and 2.1 %), sensitivity (QL < 25 ng), and accuracy (recovery between 93 % and 102 %). It was successfully applied to determine total sialic acids in a model protein drug product, demonstrating promising potential as an accurate, robust, efficient, and cost-effective approach for sialylation evaluation.

01 Sep 2025·Comparative Biochemistry and Physiology D-Genomics & Proteomics

Dynamic metabolic profiling of sea cucumbers (Apostichopus japonicus) under predation stress

Article

Author: Wang, Shuo ; Sun, Yongxin ; Song, Jian ; Qu, Liang ; Wang, Qingzhi ; Wang, Meng ; Wang, Chong ; Zhao, Chong

The present study utilized non-targeted liquid chromatography-tandem mass spectrometry (LC-MS/MS) to investigate the metabolomic responses of sea cucumber (Apostichopus japonicus) juveniles under predation stress induced by sea stars (Asterina pectinifera) at various time points (3 h, 12 h, 72 h, and 96 h). The findings revealed significant temporal changes in the metabolic profiles of the sea cucumber juveniles under predation stress, with 25, 72, 55, and 53 metabolic products exhibiting significantly different expression levels at each time point (positive ion mode, P < 0.05), respectively. Notably, the impact of predation stress was most pronounced at the 12-h mark. Multivariate statistical methods, including principal component analysis (PCA) and partial least squares-discriminant analysis (PLS-DA), further confirmed distinct clustering of the experimental group away from the control group at each time point, with the most pronounced separation occurring at 12 h, indicating a significant and time-dependent metabolic response to predation stress. Key metabolic pathways associated with predation stress were identified, such as carbon metabolism, pentose phosphate pathway, purine metabolism, riboflavin metabolism, longevity regulation, and antifolate resistance pathways, by integrating variable importance in the projection (VIP), fold change (FC), and P-value. KEGG enrichment analysis highlighted significant expression changes of key metabolites like carbamoyl phosphate, gluconolactone, inosine, 2'-deoxyguanosine, and adenylate in response to predation stress, potentially related to energy metabolism, antioxidant defense, signal transduction, and cellular stress responses. The study provides novel insights into the metabolic adaptability of sea cucumber juveniles to predation stress.

01 Aug 2025·FOOD CHEMISTRY

Sensory quality and Metabolomic fingerprinting of Lacticaseibacillus paracasei-derived fermented soymilk beverages: Impact of starter strain and storage

Article

Author: Wang, Jicheng ; Zhang, Wenyi ; Kwok, Lai-Yu ; Shi, Linbo ; Chen, Guohuan

Few previous studies have concurrently evaluated the effects of different fermentation bacterial strains and storage durations on the characteristics of fermented soymilk beverages (FSBs). This study used Lacticaseibacillus paracasei to conduct systematic assessments and demonstrated that soy protein is the optimal ingredient for sensory evaluation. Both investigated strains (PC-01 and PC646) significantly enhanced the nutritional and flavor profiles of FSBs, introducing a range of bioactive metabolites absent in non-fermented soymilk. Throughout the storage period, a decline in pH and viable bacterial counts was observed, along with an increase in titratable acidity and stability. Moreover, the metabolomic structure and metabolite abundance varied considerably between the FSBs produced by the two strains, with the non-volatile components showing greater variation, whereas the storage duration predominantly influenced the volatile metabolite components. These insights highlight the critical roles of strain selection and storage duration in shaping the nutritional and sensory qualities of FSBs.

News (Medical) associated with Aceneuramic acid

23 Mar 2017

·www.biospace.com

Bay Area’s Ultragenyx Tanks as Seizure Drug Flunks Phase II Test

March 23, 2017 By Alex Keown , BioSpace.com Breaking News Staff NOVATO, Calif. – Shares of Ultragenyx Pharmaceuticals are down more than 8 percent today after the company announced its Phase II drug UX007 to treat seizures failed to meet trial goals. In its announcement, Ultragenyx said UX007 did not reduce the frequency of number of observable seizures among patients during an eight-week period of treatment. The company said treatment with UX007 did show a reduction in absence seizures captured on EEG, but not observable seizures captured by diary. Patients treated with UX007 demonstrated a reduction of 13.4 percent in overall seizure frequency relative to placebo, the company said in its announcement. During the Phase II trial, patients kept a daily seizure diary to capture observable seizures in patients. An electroencephalography (EEG) was used at baseline and week eight to capture absence seizures. Dosing was initiated over a two-week titration period until the patient reached the target dose of 35 percent of total daily calories from UX007, the company said. UX007 is a highly purified, pharmaceutical-grade synthetic seven carbon fatty acid triglyceride intended to provide patients with medium-length, odd-chain fatty acids that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process, according to company information. The drug is being developed to treat seizures in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS is a severely debilitating disease characterized by seizures, developmental delay, and movement disorders. Glut1 DS is caused by a genetic defect in the transport of glucose into the brain. There are currently no FDA approved treatments specific to Glut1 DS. Emil D. Kakkis , president and chief executive officer of Ultragenyx , said the Phase II data showing the 13.4 percent reduction in seizures suggests UX007 has a “clinically meaningful effect in Glut1DS patients with absence seizures.” Kakkis said the company is looking forward to continue studying UX007 in Ultragenyx’s Phase III study in Glut1 DS patients with movement disorders. He said the company will continue to evaluate its options for UX007 in the indication for seizures. There were no deaths and no treatment-related serious adverse events during the trial. Two of the 36 enrolled patients discontinued treatment during the eight-week placebo-controlled period. During the trial, 12 patients discontinued use during the extension period. Two patients discontinued due to adverse events, four patients due to tolerability reasons and eight for compliance or study burden issues, the company said. Ultragenyx could get a rebound if news is positive regarding its Phase III enzyme replacement therapy candidate rhGUS for the treatment of Mucopolysaccharidosis 7. In July, the company announced the late-stage study met its primary endpoint of reducing urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment. Ultragenyx is set to seek regulatory approval with the U.S. Food and Drug Administration and the European Medicines Agency later this year. In November, Ultragenyx was forced to shelve plans for a quick approval of its investigational Aceneuramic Acid Prolonged Release (Ace-ER) for the treatment of adult patients with GNE Myopathy. The company announced it withdrew a conditional Marketing Authorization Application after a Phase II study “did not provide a sufficient amount of evidence to support an approval” at the time it was received. The company has launched a Phase III trial and plans to submit another MAA to the EMA after data is available later this year.

Phase 2Phase 3Clinical Result

20 Jan 2017

·www.bioportfolio.com

Ultragenyx Pharmaceutical Inc RARE Pharmaceuticals Healthcare Deals and Alliances Profile Prices from USD $250

Summary Ultragenyx Pharmaceutical Inc Ultragenyx is a biotechnology company that develops lifeenhancing therapeutics for rare and ultrarare genetic diseases. The company's products include KRN23, rhPPCA, rhGUS, UX007 and Aceneuramic acid. Its Aceneuramic acid is developed for multiple rare genetic disorders. Ultragenyx develops recombinant fully human monoclonal IgG1 antibody, KRN23 to treat bone disease XLinked Hypophosphatemia and tumorinduced osteomalacia. The company also develops investigational enzyme replacement therapies, recombinant human betaglucuronidase for the development for Mucopolysaccharidosis 7 and rhPPCA for treatment of galactosialidosis. It partners with academic institutes and companies. Ultragenyx is headquartered in Novato, California, the US. Ultragenyx Pharmaceutical Inc RARE Pharmaceuticals Healthcare Deals and Alliances Profile provides you comprehensive data and trend analysis of the company's Mergers and Acquisitions MAs, partnerships and financings. The report provides detailed information on Mergers and Acquisitions, Equity/Debt Offerings, Private Equity, Venture Financing and Partnership transactions recorded by the company over a five year period. The report offers detailed comparative data on the number of deals and their value categorized into deal types, subsector and regions. GlobalData derived the data presented in this report from proprietary inhouse Pharma eTrack deals database, and primary and secondary research. Scope Financial Deals Analysis of the company's financial deals including Mergers and Acquisitions, Equity/Debt Offerings, Private Equity, Venture Financing and Partnerships. Deals by Year Chart and table displaying information encompassing the number of deals and value reported by the company by year, for a five year period. Deals by Type Chart and table depicting information including the number of deals and value reported by the company by type such as Mergers and Acquisitions, Equity/Debt Offering etc. Deals by Region Chart and table presenting information on the number of deals and value reported by the company by region, which includes North America, Europe, Asia Pacific, the Middle East and Africa and South and Central America. Deals by Subsector Chart and table showing information on the number of deals and value reported by the company, by subsector. Major Deals Information on the company's major financial deals. Each such deal has a brief summary, deal type, deal rationale; and deal financials and target Company's major public companies key financial metrics and ratios. Business Description A brief description of the company's operations. Key Employees A list of the key executives of the company. Important Locations and Subsidiaries A list and contact details of key centers of operation and subsidiaries of the company. Key Competitors A list of the key competitors of the company. Key Recent Developments A brief on recent news about the company. Reasons to Buy Get detailed information on the company's financial deals that enable you to understand the company's expansion/divestiture and fund requirements The profile enables you to analyze the company's financial deals by region, by year, by business segments and by type, for a five year period. Understand the company's business segments' expansion / divestiture strategy The profile presents deals from the company's core business segments' perspective to help you understand its corporate strategy. Access elaborate information on the company's recent financial deals that enable you to understand the key deals which have shaped the company Detailed information on major recent deals includes a summary of each deal, deal type, deal rationale, deal financials and Target Company's key financial metrics and ratios. Equip yourself with detailed information about the company's operations to identify potential customers and suppliers. The profile analyzes the company's business structure, locations and subsidiaries, key executives and key competitors. Stay uptodate on the major developments affecting the company Recent developments concerning the company presented in the profile help you track important events. Gain key insights into the company for academic or business research Key elements such as break up of deals into categories and information on detailed major deals are incorporated into the profile to assist your academic or business research needs. Note*: Some sections may be missing if data is unavailable for the company.

AntibodyAcquisition

100 Deals associated with Aceneuramic acid

External Link

KEGG	Wiki	ATC	Drug Bank
D11016	Aceneuramic acid	M09AX05	DB11797

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Distal Myopathy, Nonaka Type	Japan	Nobelpharma Co., Ltd.	26 Mar 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Thrombocytopenia	Phase 2	-	Hadassah University Hospital	01 Sep 2016
Acute Lung Injury	Preclinical	China	Central South University of Forestry & Technology	20 Sep 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04671472 (phase III, Pubmed \| Orphanet J Rare Dis)	Phase 3	Distal Myopathy, Nonaka Type mutations in the GNE gene	14	Aceneuramic acid (SA-ER)	ykylamsdqe(gpnffycmcl): LSM difference = 2.51	Positive	11 Aug 2023
				Placebo
NCT02377921 (UX001-CL301, Pubmed \| Ann Neurol \| Neurology) Manual	Phase 3	Distal Myopathy, Nonaka Type	89	aceneuramic acid	hypunawswh(pemiavwcuy) = wpazxrynfb rnfpwrphhn (yzzbofsiya )	Negative	30 Apr 2019
				Placebo	hypunawswh(pemiavwcuy) = bomrhwdixj rnfpwrphhn (yzzbofsiya )
NCT02731690 (CTgov)	Phase 2	Distal Myopathy, Nonaka Type	42	UX001 (UX001 6g/Day)	jzfamucjxz = ntrzjtoebr gaeiuedtvi (bpwtrvirsa, blfuxjqwnl - mxsmjpgsqz) View more	-	19 Feb 2019
				Ace-ER (Ace-ER 6 g/Day)	jvnpflioje(qhnquttufj) = hmlgxpnuha pkxdjgswhx (yuvoowdvyq, ftxizdydcy - xdzfvatpag) View more
NCT02736188 (CTgov)	Phase 3	Distal Myopathy, Nonaka Type	143	Ace-ER (Ace-ER 6 g/Day)	sfunnluouo = orkqmczria rlfqxodjnf (iavigltiky, okeuxlpdpu - ykrzdchhns) View more	-	19 Feb 2019
				Ace-ER (Ace-ER 6 g/Day (Parent Study Treatment: Ace-ER 6 g/Day))	jeyxsepzdt(lvjqvoejyw) = earmfcxvrd pxemzevriv (vgupjhmvzh, kbyhqcqytw - wnrsoxlecf) View more
NCT02377921 (UX001-CL301 \| GNEM, CTgov)	Phase 3	Distal Myopathy, Nonaka Type	89	Placebo	ukhnpnhaqy(tvodyvopry) = isyekwbpya hcmcmzkfnd (pntdkydxya, drrkgpfbnd - gguwntethm) View more	-	09 Jul 2018
NCT01830972 (CTgov)	Phase 2	Distal Myopathy, Nonaka Type	59	SA-IR 500 mg (Crossover Participants)	eblpvldylp = bjdvijopkm hbcavrdsco (cmifpkrmfq, ahfqwyzlnh - exlewzqyml) View more	-	13 Mar 2018
				SA-IR 500 mg (Naïve Participants)	mizoqgfueb = gljcdmqzpj qsropfcpdd (fiwjeyenjg, shixlcrfuy - jbtwvzqjmn) View more
AAI2017	Not Applicable	-	-	Sialic acid (Clostridium perfringens neuraminidase (NA))	wmobzzeeqa(ibznlwkpyg) = bcsvumcvwv ekltlqmctv (ybpthmusau )	-	01 May 2017
				Sialic acid (Human tumor necrosis factor (hTNF))	wmobzzeeqa(ibznlwkpyg) = bnttmtquom ekltlqmctv (ybpthmusau )

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