RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China), Orphan Drug (United Kingdom)

Structure/Sequence

Molecular FormulaC50H78N4O14

InChIKeyKUBARPMUNHKBIQ-VTHUDJRQSA-N

CAS Registry928659-70-5

Clinical Trials associated with Eliglustat

NCT06558773

/ RecruitingPhase 2IIT

GSL Synthetase Inhibitor in Combination With Immune Checkpoint Inhibitor and/or Regorafenib for Patients With Advanced/Metastatic pMMR/MSS Colorectal Cancer:an Open-Label, Randomized,Phase II Study

In this single-center,open-label, randomized, phase II study, the efficacy and feasibility of GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib therapeutic regimen will be evaluated in patients with advanced/metastatic proficient mismatch repair/microsatellite stable (pMMR/MSS) colorectal cancer (CRC).In this clinical trial, a total of 120 eligible patients were stratified randomly (with/without liver metastases) assigned to the 3 arms in a 1:1:1 ratio: comparator group-arm A (Regorafenib+Immune checkpoint inhibitor) ,experimental group-arm B (Eliglustat+Immune checkpoint inhibitor) and experimental group-arm C (Eliglustat+Immune checkpoint inhibitor+Regorafenib).It aims to: 1).assess the antitumor effects of GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib;2).evaluate the immunological or clinical predictive biomarkers for efficacy and toxicity; 3).detect the transformation of tumor microenvironment (TME) and dynamic changes of immune cells in peripheral blood after the treatment with GSL synthetase inhibitor in combination with immune checkpoint inhibitor and/or regorafenib.

Start Date01 Mar 2026

Sponsor / Collaborator

Chinese People's Liberation Army General Hospital

NCT07090538

/ Not yet recruitingEarly Phase 1IIT

A Single-arm Exploratory Trial of GSL Synthetase Inhibitor Plus Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and/ or Immune Checkpoint Inhibitor in Advanced/Metastatic Neuroblastoma.

This exploratory clinical study will evaluate the efficacy and feasibility of combining a GSL synthase inhibitor with a granulocyte-macrophage colony stimulating factor (GM-CSF) in patients with advanced or metastatic neuroblastoma. Six to eight eligible patients are expected to be treated in this clinical trial: 1) Assessing the anti-tumour effects of GSL synthase inhibitors in combination with immune checkpoint inhibitors and/or GM-CSF; 2) To assess immunological or clinical predictive biomarkers of efficacy and toxicity; and 3) Detecting changes in the tumour microenvironment (TME) and the dynamics of peripheral blood immune cells after treatment with a GSL synthase inhibitor combined with GM-CSF.

Start Date01 Aug 2025

Sponsor / Collaborator

Chinese People's Liberation Army General Hospital

CTRI/2024/08/072953

/ RecruitingPhase 3

A Multicenter, Open-Label, Single-Arm,Three-Cohort, Phase 3, Clinical Study toEvaluate the Efficacy, Safety andPharmacokinetics of Eliglustat SublingualFilm in Patients with Gaucher Disease Type 1and Type 3. - NIL

Start Date02 Sep 2024

Sponsor / Collaborator

Amneal Pharmaceuticals Pvt Ltd.

100 Clinical Results associated with Eliglustat

100 Translational Medicine associated with Eliglustat

100 Patents (Medical) associated with Eliglustat

175

Literatures (Medical) associated with Eliglustat

01 Jun 2026·MOLECULAR GENETICS AND METABOLISM

Repurposing Gaucher disease therapy for Saposin C deficiency: Proof-of-concept with eliglustat

Review

Author: Deegan, Patrick B ; Minea, Carmen

Saposin C (Sap C) deficiency (GDSAPC, OMIM #610539, ORPHA:309252) is an ultra-rare autosomal recessive disorder caused by mutations in the PSAP gene. Sap C functions as an essential activating cofactor of glucosylceramidase (GCase) and facilitates the degradation of glucosylceramide in the lysosome. In the absence of its activator, GCase is structurally intact but its function is impaired, leading to pathological accumulation of glucosylceramide in the lysosome. Sap C deficiency resembles another rare autosomal recessive disorder, Gaucher disease (GD), in which pathogenic variants in the GBA1 gene result in GCase deficiency and accumulation of glucosylceramide within the lysosomal compartment of the mononuclear phagocyte system. Despite the overlapping clinical features of Sap C and GD, the therapeutic implication is utterly different. To date, no specific therapy is approved for Sap C deficiency. Enzyme replacement therapy (ERT), highly effective in GD is biologically implausible in Sap C deficiency, where the GCase structure is normal but lacks its essential activator for its functions. In GD, substrate reduction therapy (SRT) is an alternative to ERT by limiting the synthesis of glucosylceramide through inhibition of glucosylceramide synthase. Given that substrate accumulation is the common pathological consequence in both GD and Sap C deficiency, we hypothesized that SRT with Eliglustat, a potent glucosylceramide synthase inhibitor, could also benefit patients with Sap C deficiency. We describe the first documented therapeutic attempt using eliglustat in Sap C deficiency. A 47-year-old patient with PSAP mutations causing Sap C deficiency who presented with features similar to those seen in GD, has received Eliglustat over the course of 9 years, demonstrating an improvement in her hepatosplenomegaly, haematological parameters, biomarkers and bone density, providing proof-of-concept that Eliglustat can be of benefit when the GCase cofactor is deficient. However, no improvement was observed in the patient's seizure activity where future brain penetrant molecules may be of benefit.

26 May 2026·Zhonghua yi xue za zhi

[Efficacy and safety of eliglustat tartrate in adults with Gaucher disease type 1].

Article

Author: Hu, L Y ; Ren, Y S ; Meng, Y ; Zhang, L ; Yang, R

To evaluate the efficacy and safety of eliglustat tartrate in adult patients with Gaucher disease type 1 (GD1). Adult patients with GD1 confirmed at the First Medical Center of Chinese People's Liberation Army General Hospital were prospectively enrolled from October 2023 to June 2024. They received eliglustat tartrate capsules treatment (oral, 84 mg, twice daily) for 52 weeks. The changes in glucosylsphingosine (Lyso-GL1), hemoglobin and platelet count, liver and spleen volumes, multiples of normal (MN) of liver and spleen volumes were observed before and after treatment, and adverse reactions during treatment were recorded. A total of 5 adult patients with GD1 type were included, and all were identified as extensive metabolizers (EMs) via CYP2D6 genotyping. Following 52 weeks of treatment, Lyso-GL1 levels decreased apparently from baseline. Hemoglobin levels and platelet count also increased. Both liver and spleen volumes decreased, with corresponding reductions in MN. One patient reported mild nausea and anorexia, which resolved spontaneously within 2 d without other adverse events. Eliglustat tartrate demonstrates favorable efficacy and safety in these 5 adult GD1patients with EMs.

01 Mar 2026·Drug Research

Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System

Article

Author: Gan, Ya ; Xia, Chunyong ; Liu, Jie

Abstract:

Both eliglustat and miglustat are substrate reduction therapies targeting glucosylceramide synthase; yet, their safety profile has not been comprehensively analyzed. This study analyzes adverse events associated with both drugs using the U.S. Food and Drug Administration Adverse Event Reporting System to provide insights for clinical safety.Adverse events were classified by MedDRA System Organ Class (SOC, v26.1). Adverse event signals were mined by disproportionality analyses, including the reporting odds ratio, the proportional reporting ratio, the multi-item gamma Poisson shrinker algorithms, and the Bayesian confidence propagation neural network.A total of 1,223 and 980 adverse event reports were retrieved from eliglustat and miglustat, respectively, involving 27 System Organ Class categories each. Some positive signals were consistent with the drug labels, including dyspepsia identified in eliglustat and diarrhoea identified in miglustat. We also identified unexpected signals not listed on the drug labels, such as paresthesia, dry skin, and ichthyosis for eliglustat and dysphagia for miglustat. For patients treated with eliglustat and miglustat, the majority of adverse events manifested more than 1 year after the initiation of therapy. Notably, male patients treated with eliglustat have the significantly higher incidence of weight increase and dry skin. Female patients treated with miglustat have the significantly higher incidence of dysphagia and cognitive disorder.In the clinical administration of eliglustat and miglustat, clinicians need to monitor the effects of adverse events varied by gender and to pay more attention to new adverse event signals.

News (Medical) associated with Eliglustat

28 May 2026

·allsci.com

Sanofi’s NDA for venglustat in Gaucher disease gets FDA priority status

Sanofi (Euronext: SAN; Nasdaq: SNY) has received priority review from the US FDA for a new drug application (NDA) for venglustat, an investigational oral glucosylceramide synthase inhibitor (GCSi), seeking approval in patients with type 3 Gaucher disease (GD3) — a rare lysosomal storage disorder with progressive neurological involvement for which no targeted pharmacological treatment currently exists in the US. The NDA seeks approval of venglustat specifically to address the neurological manifestations of GD3, including cerebellar ataxia and cognitive deficits, in adult and pediatric patients who have previously achieved stabilization of systemic disease manifestations with enzyme replacement therapy (ERT). The US FDA has assigned a target action date of November 25, 2026. Venglustat previously received breakthrough therapy designation, fast-track designation, and orphan designation in the US for GD3. A parallel regulatory review is also underway in the EU, and Sanofi said it plans to pursue additional global filings for the indication in 2026. The NDA is supported by data from the LEAP2MONO Phase III study (NCT05222906), a randomized, double-blind, double-dummy, active-comparator trial evaluating venglustat against intravenous ERT in patients aged 12 and older with GD3. Forty-three patients were randomized 1:1 to receive once-daily oral venglustat plus placebo infusion, or ERT every two weeks plus placebo tablet. Eligible patients were required to have received ERT for at least three years and to have met therapeutic goals for systemic disease control prior to enrollment. The trial’s co-primary endpoints were change from baseline to week 52 in the Scale for Assessment and Rating of Ataxia (SARA) modified total score and change in the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score. Venglustat met both primary endpoints, with a reported p-value of 0.007. The drug also met three of four key secondary endpoints. Systemic secondary endpoints included percent change in spleen volume, liver volume, platelet count, and hemoglobin levels. Biomarker endpoints assessed percent change in cerebrospinal fluid and plasma glucosylceramide (GL-1) and lyso-GL-1. Venglustat was described as generally well tolerated in the trial, with no new safety signals identified relative to earlier studies. The most commonly reported adverse events included headache (14.3% in the venglustat arm versus 18.2% in the ERT arm), nausea (14.3% versus 4.5%), spleen enlargement (14.3% versus 0), and diarrhea (14.3% versus 0). The open-label phase of LEAP2MONO is ongoing, and Sanofi said results will be presented when available. Venglustat is a brain-penetrant, oral small-molecule inhibitor of glucosylceramide synthase, the enzyme responsible for the committed step in glycosphingolipid biosynthesis. By reducing upstream substrate production, it functions as a substrate reduction therapy (SRT), a mechanistic class that also includes the approved agents eliglustat (Cerdelga) and miglustat (Zavesca). Neither of those agents, however, was developed or approved for GD3, and neither demonstrates meaningful CNS penetration — a critical limitation given that the neurological manifestations of GD3 are driven by glycosphingolipid accumulation in the central nervous system. ERT, the current clinical standard for GD3, addresses systemic features such as organomegaly, cytopenias, and bone disease, but does not cross the blood-brain barrier and has no demonstrated effect on neurological progression. In January 2026, the US FDA expanded the label for imiglucerase (Cerezyme), also a Sanofi product, to include a GD3 indication — but that approval was based on systemic outcomes data from an observational registry and does not address the neurological dimension of the disease. If approved, venglustat would represent the first US-authorized therapy specifically indicated for the neurological manifestations of GD3, filling a gap that has persisted throughout the decades-long history of Gaucher disease treatment. The filing adds to a competitive landscape in which no other late-stage CNS-directed agent for GD3 is currently identified in the public domain, positioning venglustat as a potential first-mover in a narrow but clinically distinct patient population. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

Phase 3Clinical ResultBreakthrough TherapyFast TrackOrphan Drug

02 Feb 2026

·www.fiercebiotech.com

Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease

Venglustat was once considered as a potential \"pipeline in a pill.\"\n Sanofi’s pipeline features a number of once-hyped candidates that are struggling to deliver in the clinic, and the latest mixed phase 3 data from venglustat continue this trend.The French Big Pharma was studying venglustat, an oral, brain-penetrant glucosylceramide synthase (GCS) inhibitor, in a pair of late-stage studies of rare lysosomal storage disorders. One of these trials, called Peridot, assessed the pill in 122 patients with Fabry disease, while the Leap2Mono trial focused on 43 patients with type 3 Gaucher disease (GD3).The Peridot study missed its primary endpoint of demonstrating superiority in patient-reported improvement of symptoms—namely neuropathic pain in upper and lower extremities, as well as abdominal pain—compared to a placebo cohort. Sanofi explained this failure by pointing to a “reduction in neuropathic and abdominal pain … observed in both study arms.”It was better news in the Leap2Mono study, where the 21 patients who received venglustat saw statistically significant improvements in neurological symptoms at Week 52 as measured by scales for ataxia and for neuropsychological status. The improvement between the venglustat cohort and the 22 patients who received enzyme replacement therapy (ERT) worked out as a p-value of 0.007, Sanofi explained in its Feb. 2 release.When it came to non-neurological secondary endpoints like changes in spleen volume, liver volume and hemoglobin level, venglustat “performed as well as ERT,” according to the pharma.Venglustat was well tolerated, Sanofi said. The most common adverse events were headache, nausea, spleen enlargement and diarrhea—although reports of headache were higher in the ERT cohort.Based on the Leap2Mono data, Sanofi said it will seek regulatory approval for venglustat in GD3. The company already markets the ERT Cerezyme and the oral glucosylceramide synthase inhibitor Cerdelga for the disease.The failure of the Peridot study doesn’t mark the end of Sanofi’s Fabry ambitions, either. As well as its approved Fabry ERT Fabrazyme, Sanofi is still running a phase 3 study of venglustat on left cardiac ventricular mass index in patients with the disease.“These findings underscore Sanofi\'s commitment to rare disease research and the promise we aim to deliver for people living with these conditions,” Sanofi’s head of R&D Houman Ashrafian, Ph.D., said in this morning’s release.“What excites us most is the potential to address critical unmet medical needs,” Ashrafian added. “A daily pill could make a serious difference for Gaucher patients facing neurological challenges.”This morning’s phase 3 readouts continued a run of mixed data for venglustat, which had at one point been hailed as one of Sanofi\'s top prospects and a potential “pipeline in a pill” contender. Those ambitions were blunted as far back as 2021, when venglustat failed midstage studies in both Parkinson’s disease and autosomal dominant polycystic kidney disease. By 2024, venglustat’s options were narrowed down to just Fabry disease and GD3 after Sanofi gave up on GM2 gangliosidosis as an indication “based on the absence of positive trends on clinical endpoints.”While Sanofi remains hopeful of getting venglustat approved for GD3, the Fabry failure this morning also continues a longer-term trend of the drugmaker’s pipeline struggling to achieve its potential. The company’s R&D chief admitted to Fierce last year that clinical setbacks for its anti-OX40L-ligand antibody amlitelimab, its oral TNF inhibitor balinatunfib and its Regeneron-partnered IL-33 candidate itepekimab had resulted in a “mixed” 2025.On an earnings call with journalists last week, Sanofi CEO Paul Hudson directly acknowledged that it had been a “bumpy ride” for R&D.“I\'m well aware of the headlines—we had some challenges,” the CEO said at the time.

$Sanofi\'s troubled GCS inhibitor fails phase 3 Fabry trial but scores in Gaucher disease$

Phase 3Clinical ResultDrug Approval

02 Feb 2026

·firstwordpharma.com

Sanofi posts a win for venglustat in Gaucher study, but a miss in Fabry disease

Sanofi announced Monday mixed results from a pair of pivotal studies of venglustat in two rare lysosomal storage disorders, with the oral glucosylceramide synthase inhibitor (GCSi) recording a hit in type 3 Gaucher disease (GD3) and a miss in Fabry disease. The contrasting outcomes are the latest to emerge from the company's late-stage pipeline, following differing results earlier this year from the investigational OX40-ligand-blocking monoclonal antibody amlitelimab in two atopic dermatitis trials.The Phase III LEAP2MONO study randomised 43 adult and paediatric patients with neurological manifestations of GD3 to receive venglustat once-daily or enzyme replacement therapy (ERT). The trial's primary endpoints were change in Scale for Assessment and Rating of Ataxia (SARA) modified total score and change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score, with key secondary goals including percent change in spleen volume, liver volume and platelet count, as well as change in haemoglobin levels.Results showed that venglustat demonstrated statistically significant improvements in neurological symptoms as measured by both SARA and RBANS at week 52 compared to ERT. Sanofi added that the therapy also achieved three out of the four key secondary endpoints, performing as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume and haemoglobin levels.Regulatory filings in GD3"What excites us most is the potential to address critical unmet medical needs. A daily pill could make a serious difference for Gaucher patients facing neurological challenges," said Houman Ashrafian, head of R&D at Sanofi. The company indicated that based on the results, it will pursue global regulatory filings for venglustat in GD3.Sanofi currently markets the injectable therapy Cerezyme (imiglucerase) for Gaucher disease, as well as the oral drug Cerdelga (eliglustat) for those with type 1 disease. The drugmaker's portfolio also includes the injectable therapy Fabrazyme (agalsidase beta) for Fabry disease. However, the latest results dent its hopes of adding an oral Fabry disease therapy to its arsenal.Fabry setbackThe Phase III PERIDOT study randomised 122 patients aged 16 years and older with Fabry disease to receive venglustat or placebo. Sanofi noted that the trial failed to hit its primary endpoint of percent change from baseline on patient-defined most bothersome symptoms, as assessed by the Fabry Disease Patient-Reported Outcome (FD-PRO) instrument.According to the company, reductions in neuropathic and abdominal pain were observed in both study arms. Additional analyses of the data are ongoing, while the late-stage CARAT trial evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease is ongoing.Findings from both LEAP2MONO and PERIDOT are scheduled to be presented this week at the annual WORLDSymposium.Aside from the positive results in GD3, Venglustat, which works by reducing the abnormal accumulation of sugar-and-fat molecules in cells and organs, has racked up a number of study failures. In 2024, Sanofi disclosed that the Phase III AMETHIST trial for GM2 gangliosidosis was discontinued based on “the absence of positive trends” for clinical endpoints, adding to a pivotal Phase II/III study setback in autosomal dominant polycystic kidney disease and another in Parkinson's disease with GBA mutations.

Phase 3Clinical Result

100 Deals associated with Eliglustat

External Link

KEGG	Wiki	ATC	Drug Bank
D09894	Eliglustat	A16AX10	DB09039

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Gaucher Disease	Japan	Genzyme Corp.	26 Mar 2015
Gaucher Disease	Japan	Sanofi KK	26 Mar 2015
Gaucher Disease, Type 1	United States	Genzyme Corp.	19 Aug 2014

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Gaucher Disease, Type 3	Phase 3	Japan	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Argentina	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Canada	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	France	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Italy	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Russia	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Spain	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Sweden	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	Turkey	Sanofi	11 Apr 2018
Gaucher Disease, Type 3	Phase 3	United Kingdom	Sanofi	11 Apr 2018

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03485677 (ELIKIDS, SSIEM2023)	Phase 3	Gaucher Disease First line CYP2D6 extensive metabolizers \| N370S variant	57	Eliglustat monotherapy	vwvpdtsrka(rotjfhbbyr) = mcdyysholf vgbopyltzf (iyefnvchmh ) View more	-	30 Aug 2023
				Eliglustat plus imiglucerase combination therapy	vwvpdtsrka(rotjfhbbyr) = faounxqyun vgbopyltzf (iyefnvchmh ) View more
ICGG Gaucher Registry (SSIEM2022)	Not Applicable	First line CYP2D6-metabolizer status	-	eliglustat	juoylrnuda(unammdbufx) = wemfczwtgm lvoghxixea (cjavwujnms ) View more	-	30 Aug 2022
				eliglustat	juoylrnuda(unammdbufx) = bmfbvedehw lvoghxixea (cjavwujnms ) View more
NCT02536755 (EXOSKEL, CTgov)	Phase 3	Gaucher Disease	31	Eliglustat, GZ385660	bccspxjntx = eshskbtgcp puqkikhoye (olqhgqgcvd, vvodgkumzp - ncvlegveig) View more	-	15 Jul 2022
NCT00891202 (ENGAGE, Pubmed \| Am J Hematol)	Phase 3	Gaucher Disease, Type 1	40	Eliglustat	rejcopkyhq(qhgtmrzbgh) = btujlzxkto xuhlpjibrl (xfmbrwauvc ) View more	Positive	23 Jun 2021
				Placebo	outuvrngwa(kmmmyeaopj) = mevronasgn emlccnpfyl (nwlevzakkz ) View more
EHA2019	Not Applicable	Gaucher Disease, Type 1 First line	-	Eliglustat	hrpvwdmspr(xngwpfrpyy) = yxtqufhguk gokbqtxzvd (cosryjrkjv ) View more	-	14 Jun 2019
NCT00358150 (Pubmed \| Am J Hematol) Manual	Phase 2	Gaucher Disease, Type 1 First line	26	eliglustat	xqgmijserh(rfcpexvehb) = gpmfbluutq ulzgzvbjcd (munbtcwovn ) View more	Positive	01 Jan 2019
NCT01074944 (EDGE, Pubmed) Manual	Phase 3	Gaucher Disease, Type 1	170	eliglustat (once-daily)	drnieqioff(gulagwotfk) = kewpwnjggi ghcpcmzkdk (ijwxcquvvc, 67.6 - 89.8)	Positive	01 Mar 2018
				eliglustat (twice-daily)	drnieqioff(gulagwotfk) = nbutaacnvj ghcpcmzkdk (ijwxcquvvc, 71.0 - 91.6)
NCT01074944 (EDGE, CTgov)	Phase 3	Gaucher Disease, Type 1	170	PAP+Eliglustat (PAP, Eliglustat: Once Daily)	zwtjzzfpch = rvhugtvsyi sjrsidylkw (losyjagnrv, oydzomamhj - yfthoayhlf) View more	-	02 Dec 2016
				PAP+Eliglustat (PAP, Eliglustat: Twice Daily)	zwtjzzfpch = rivhacjafd sjrsidylkw (losyjagnrv, aabejsjuqd - jbtwlsbbtr) View more
NCT00943111 (ENCORE, Pubmed \| Lancet) Manual	Phase 3	Gaucher Disease, Type 1	160	Eliglustat	pzwdrkmgeg(hucyimvynt) = epfzobyepd uzwyyzxxuc (xqsexyvmbm ) View more	Non-superior	13 Jun 2015
				imiglucerase	pzwdrkmgeg(hucyimvynt) = iemcncaplx uzwyyzxxuc (xqsexyvmbm ) View more
NCT00891202 (ENGAGE, Pubmed \| J Coll Physicians Surg Pak \| JAMA) Manual	Phase 3	Splenomegaly \| Gaucher Disease, Type 1	40	eliglustat	mbcuzeooyl(rhwmbcuxgz) = lqitzgtmox ugjwasofvm (yvwoozvtqs, -32.57 to -22.97)	Positive	17 Feb 2015
				Placebo	mbcuzeooyl(rhwmbcuxgz) = kjmksycbjm ugjwasofvm (yvwoozvtqs, -2.54 to 7.06)

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