Delving into the Latest Updates on Aceneuramic acid with Synapse

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

NCT02845609 / CompletedPhase 2IIT

A Phase 2 Study to Evaluate the Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Related Thrombocytopenia

Sialic Acid-Extended Release (SA-ER, aceneuramic acid, UX001) is an extended release formulation of sialic acid (SA, also known as N-acetylneuraminic acid or NANA). The SA-ER is currently studied as a substrate replacement therapy for patients with GNE myopathy. The investigators plan to study the SA-ER compound in a cohort of five patients with GNE-related thrombocytopenia.

Start Date01 Sep 2016

Sponsor / Collaborator

Hadassah University Hospital

100 Clinical Results associated with Aceneuramic acid

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100 Translational Medicine associated with Aceneuramic acid

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100 Patents (Medical) associated with Aceneuramic acid

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265

Literatures (Medical) associated with Aceneuramic acid

01 Nov 2024·Journal of Neurology, Neurosurgery & Psychiatry

Safety and efficacy of aceneuramic acid in GNE myopathy: open-label extension study

Letter

Author: Nakamori, Masayuki ; Oya, Yasushi ; Tateyama, Maki ; Takahashi, Masanori P ; Kuroda, Hiroshi ; Nishino, Ichizo ; Yamada, Shinichiro ; Hashizume, Atsushi ; Izumi, Rumiko ; Suzuki, Naoki ; Yamashita, Satoshi ; Katsuno, Masahisa ; Kato, Masaaki ; Mori-Yoshimura, Madoka ; Warita, Hitoshi ; Aoki, Masashi ; Asada, Ryuta ; Yamaguchi, Takuhiro

01 Sep 2024·Journal of Attention Disorders

Extended-Release Mixed Amphetamine Salts for Comorbid Adult Attention-Deficit/Hyperactivity Disorder and Cannabis Use Disorder: A Pilot, Randomized Double-Blind, Placebo-Controlled Trial

Article

Author: Mariani, John J ; Brezing, Christina A ; Choi, C Jean ; Mahony, Amy L ; Brooks, Daniel J ; Basaraba, Cale ; Naqvi, Nasir ; Pavlicova, Martina ; Levin, Frances R

Objective: To determine if treatment of co-occurring adult ADHD and Cannabis Use Disorder (CUD) with extended-release mixed amphetamine salts (MAS-ER) would be effective at improving ADHD symptoms and promoting abstinence. Method: A 12-week randomized, double-blind, two-arm pilot feasibility trial of adults with comorbid ADHD and CUD ( n = 28) comparing MAS-ER (80 mg) to placebo. Main outcomes: ADHD: ≥30% symptom reduction, measured by the Adult ADHD Investigator Symptom Rating Scale (AISRS). CUD: Abstinence during last 2 observed weeks of maintenance phase. Results: Overall, medication was well-tolerated. There was no significant difference in ADHD symptom reduction (MAS-ER: 83.3%; placebo: 71.4%; p = .65) or cannabis abstinence (MAS-ER: 15.4%; placebo: 0%; p = .27). MAS-ER group showed a significant decrease in weekly cannabis use days over time compared to placebo ( p < .0001). Conclusions: MAS-ER was generally well-tolerated. The small sample size precluded a determination of MAS-ER’s superiority reducing ADHD symptoms or promoting abstinence. Notably, MAS-ER significantly reduced weekly days of use over time.

15 Aug 2024·Clinical Cancer Research

Urinary Metabolite Diagnostic and Prognostic Liquid Biopsy Biomarkers of Lung Cancer in Nonsmokers and Tobacco Smokers

Article

Author: Pine, Sharon R ; Dalal, Bhavik ; Khan, Mohammed ; Toulabi, Leila ; Parker, Amelia L ; Patel, Daxesh P ; Harris, Curtis C ; Krausz, Kristopher W ; Bowman, Elise D ; Gonzalez, Frank J ; Haznadar, Majda ; Tada, Takeshi ; Robles, Ana I ; Oike, Takahiro ; Kanke, Yasuyuki

AbstractPurpose:Nonsmokers account for 10% to 13% of all lung cancer cases in the United States. Etiology is attributed to multiple risk factors including exposure to secondhand smoking, asbestos, environmental pollution, and radon, but these exposures are not within the current eligibility criteria for early lung cancer screening by low-dose CT (LDCT).Experimental Design:Urine samples were collected from two independent cohorts comprising 846 participants (exploratory cohort) and 505 participants (validation cohort). The cancer urinary biomarkers, creatine riboside (CR) and N-acetylneuraminic acid (NANA), were analyzed and quantified using liquid chromatography–mass spectrometry to determine if nonsmoker cases can be distinguished from sex and age-matched controls in comparison with tobacco smoker cases and controls, potentially leading to more precise eligibility criteria for LDCT screening.Results:Urinary levels of CR and NANA were significantly higher and comparable in nonsmokers and tobacco smoker cases than population controls in both cohorts. Receiver operating characteristic analysis for combined CR and NANA levels in nonsmokers of the exploratory cohort resulted in better predictive performance with the AUC of 0.94, whereas the validation cohort nonsmokers had an AUC of 0.80. Kaplan–Meier survival curves showed that high levels of CR and NANA were associated with increased cancer-specific death in nonsmokers as well as tobacco smoker cases in both cohorts.Conclusions:Measuring CR and NANA in urine liquid biopsies could identify nonsmokers at high risk for lung cancer as candidates for LDCT screening and warrant prospective studies of these biomarkers.

1

News (Medical) associated with Aceneuramic acid

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.

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100 Deals associated with Aceneuramic acid

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External Link

KEGG	Wiki	ATC	Drug Bank
D11016	Aceneuramic acid	M09AX05	DB11797

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Distal Myopathy, Nonaka Type	JP	Nobelpharma Co., Ltd.	-

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Distal Myopathy, Nonaka Type	NDA/BLA	BG	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	FR	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	IT	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	CA	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	IL	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	US	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Distal Myopathy, Nonaka Type	NDA/BLA	GB	Ultragenyx Pharmaceutical, Inc.	20 May 2015
Thrombocytopenia	Discovery	-	Hadassah University Hospital	01 Sep 2016

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Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02377921 (UX001-CL301, Pubmed \| Ann Neurol) Manual	Phase 3	Distal Myopathy, Nonaka Type	89	aceneuramic acid	(zmrwjqupfg) = xvkgjnvodz uzctvuxdgu (evsvmcbxkb )	Negative	30 Apr 2019
				Placebo	(zmrwjqupfg) = hcgteswdga uzctvuxdgu (evsvmcbxkb )
NCT02731690 (CTgov)	Phase 2	Distal Myopathy, Nonaka Type	42	UX001 (UX001 6g/Day)	(zvfnldkrzy) = omnsuesvff vwcpmcdtsb (lnbudzozuq, bquetddfay - nkcbytkdnc) View more	-	19 Feb 2019
				Ace-ER (Ace-ER 6 g/Day)	fuqnyffvri(spafcgtxcd) = lbjhyiblel orlotyohgn (twnkwudmik, pbywnsoori - fodtkmazkw) View more
NCT02377921 (UX001-CL301 \| GNEM, CTgov)	Phase 3	Distal Myopathy, Nonaka Type	89	Placebo	cumdgeepae(agewgfczkp) = vvhfzufvyx airmdixjte (jwfvqyejav, cimuxvjpec - egtuesakni) View more	-	09 Jul 2018
NCT01830972 (CTgov)	Phase 2	Distal Myopathy, Nonaka Type	59	SA-IR 500 mg (Crossover Participants)	swvuztjoof(ttgvxktecn) = twlgeipwce yacirymjxz (paswmgycml, xlobtvvwsa - yzkuqzlrvs) View more	-	13 Mar 2018
				SA-IR 500 mg (Naïve Participants)	hvlfnnbgxe(mbdjhslehr) = ahsjuhzjax kpfofkojzx (yeanprchbu, bdtxlhmbvs - fudzexbgtl) View more
AAI2017	Not Applicable	-	-	Sialic acid (Clostridium perfringens neuraminidase (NA))	npoeaykeyo(vdcwnaqkrp) = syiclopgda xfrqmfnkqy (pwhxqnealk )	-	01 May 2017
				Sialic acid (Human tumor necrosis factor (hTNF))	npoeaykeyo(vdcwnaqkrp) = leikznlpsl xfrqmfnkqy (pwhxqnealk )
ASCO2004	Phase 2	Extensive stage Small Cell Lung Cancer	30	ACE chemotherapy + pegfilgrastim	(pqktxpfval) = pcnfbgaztx ywumetbwsa (gtcaswvnwm ) View more	-	15 Jul 2004