RegulationPriority Review (US), Fast Track (US), Accelerated Approval (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), Priority Review (CN), Orphan Drug (JP), SAKIGAKE (JP), Conditional marketing approval (JP)

Gene Sequence

Sequence Code 29547660

Source: *****

Clinical Trials associated with Viltolarsen

CTR20213302 / Active, not recruitingPhase 2

一项在能走动和不能走动的杜氏肌营养不良症（DMD）男童患者中评价Viltolarsen与自然史对照相比的安全性、耐受性和有效性的II 期、开放标签研究

[Translation] A Phase II, open-label study evaluating the safety, tolerability, and efficacy of viltolarsen compared with natural history controls in ambulatory and nonambulatory boys with Duchenne muscular dystrophy (DMD)

主要目的：评价viltolarsen以每周80 mg/kg的剂量静脉给药(IV)治疗能走动和卧床的≥8岁DMD男孩的安全性和耐受性。次要目的：与自然病程对照相比，以每周80 mg/kg的剂量静脉给药viltolarsen48周治疗能走动和卧床的≥8岁DMD男孩的疗效。探索性目的：评价viltolarsen治疗对患者DMD健康相关生活质量的影响，评价DMD患者的咳嗽强度，评价DMD患者走动能力的保留情况，评价日常活动和睡眠-觉醒模式，以探索viltolarsen治疗对患者DMD的影响。

[Translation]

Primary objective: To evaluate the safety and tolerability of viltolarsen at a dose of 80 mg/kg intravenously (IV) weekly in the treatment of ambulatory and bedridden boys aged ≥8 years with DMD. Secondary objective: To compare the efficacy of viltolarsen at a dose of 80 mg/kg IV weekly for 48 weeks in the treatment of ambulatory and bedridden boys aged ≥8 years with DMD compared with a natural history control. Exploratory objective: To evaluate the effect of viltolarsen treatment on the health-related quality of life of patients with DMD, evaluate the cough intensity of patients with DMD, evaluate the retention of ambulatory ability of patients with DMD, evaluate daily activities and sleep-wake patterns, in order to explore the effect of viltolarsen treatment on patients with DMD.

Start Date17 Mar 2022

Sponsor / Collaborator

Hangzhou Tigermed Consulting Co., Ltd.

[+1]

EUCTR2019-002076-13-NO / Not yet recruitingPhase 3

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53

Start Date08 Sep 2021

Sponsor / Collaborator

NS Pharma, Inc.

NCT04956289 / CompletedPhase 2

A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls

This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.

Start Date01 Jul 2021

Sponsor / Collaborator

NS Pharma, Inc.

[+1]

100 Clinical Results associated with Viltolarsen

100 Translational Medicine associated with Viltolarsen

100 Patents (Medical) associated with Viltolarsen

Literatures (Medical) associated with Viltolarsen

07 Nov 2023·Journal of neuromuscular diseases

Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Letter

Author: Straub, Volker ; Mercuri, Eugenio ; Muntoni, Francesco ; Servais, Laurent

07 Nov 2023·Journal of neuromuscular diseases

Reply to F. Muntoni et al.: “In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Letter

Author: Clemens, Paula R ; Hoffman, Eric P

01 Oct 2023·Drug metabolism and disposition: the biological fate of chemicals

Prediction of Human Pharmacokinetics of Phosphorodiamidate Morpholino Oligonucleotides in Duchenne Muscular Dystrophy Patients Using Viltolarsen.

Article

Author: Mori, Jumpei ; Yamada, Tetsuhiro ; Imai, Shunji ; Sasaki, Yoshihiro ; Kusano, Kazutomi ; Suda, Yusuke

Several modified antisense oligonucleotides (ASOs) have recently been approved for clinical use. Some are phosphorodiamidate morpholino oligomers (PMOs), which, unlike other nucleic acids, are not negatively charged. Thus, PMOs differ from other ASOs in their pharmacokinetic (PK) properties. Drugs with a PMO backbone have been administered to Duchenne muscular dystrophy pediatric patients; however, appropriate methodologies are not currently available to predict their human PK from nonclinical data. In this study, we used viltolarsen as a representative PMO to investigate the applicability of the allometric scaling approach to human PK prediction. We first summarized the nonclinical and clinical PK data for viltolarsen as showing high total clearance, low serum protein binding, metabolic resistance, and urinary excretion as the unchanged drug in both animals and humans. We then investigate the PK of viltolarsen in mice, rats, cynomolgus monkeys, and dogs and used the results, with body weight, to extrapolate to humans by several methods. The estimate of human total clearance obtained from cynomolgus monkeys was the best, and body weight may be the key factor in accurately predicting human total clearance. In contrast, all of the well-known prediction methods for the volume of distribution at steady state gave underestimates. However, the human PK profiles predicted from the PK parameters in cynomolgus monkeys fit the observed human plasma concentrations well. These results are expected to contribute to the further development of PMOs. SIGNIFICANCE STATEMENT: We investigated how to predict the human PK of phosphorodiamidate morpholino oligomers from nonclinical data. The estimates of human PK parameters and profiles determined from cynomolgus monkeys by an allometric scaling approach were the most suitable, and the cynomolgus monkey body weight may be the key factor in accurately predicting human total clearance.

News (Medical) associated with Viltolarsen

17 Jun 2024

·www.prnewswire.com

Pfizer's Setback in Duchenne Muscular Dystrophy Treatment Trial Clears Path for Sarepta, and Many Other Pharma Companies | DelveInsight

Pfizer's mini-dystrophin gene therapy fordadistrogene movaparvovec failure in the Duchenne muscular dystrophy treatment opens new opportunities for several pharmaceutical companies such as Sarepta Therapeutics (SRP-5051), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to advance their therapies and potentially dominate the DMD market. LAS VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne muscular dystrophy is the most prevalent type of muscular dystrophy found in children. This genetic condition leads to ongoing muscle weakening and degeneration. It is one among nine different forms of muscular dystrophy. According to DelveInsight's estimates, the US accounted for around ~17K total prevalent cases of Duchenne muscular dystrophy in 2023. The highest cases were found in the children of the 5-9 age group, these numbers are expected to grow by the year 2034. As per DelveInsight's analysis, the most commonly associated comorbidities accompanied with DMD included the highest cases of scoliosis accounting for around ~20% of cases followed by ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting for around ~19%, ~18%, and ~15% cases respectively in the US in the year 2023. The standard of care for DMD typically involves a combination of corticosteroids and surgical interventions. Prednisone and deflazacort are commonly prescribed glucocorticoids that have been utilized for over two decades to increase muscular strength in DMD patients. Surgery may be necessary to address contractures, spinal curvature, or scoliosis, which can affect breathing and overall mobility. Additionally, interventions such as pacemakers or other cardiac devices may be utilized to improve heart function. Sarepta Therapeutics is a key player in the DMD market, boasting three approved assets and two in the current pipeline. Despite its dominance, emerging competition is evident. The historical success rate for market entry varies across the 7MM. Notably, the DMD market exhibits geographic disparities in regulatory approvals, with instances of products approved in one jurisdiction and rejected in another, despite identical clinical data, reflecting the distinct standards of regulatory bodies such as EMA and FDA. Learn more about the FDA-approved DMD drugs @ Drugs for Duchenne Muscular Dystrophy Treatment The current US DMD treatment market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with DMD. In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat DMD patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany. ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta's gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process. In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting DMD patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan, the only approved treatment is VILTEPSO (viltolarsen). ELEVIDYS is a prescription gene therapy designed for ambulatory children aged 4 to 5 years with DMD and a confirmed dystrophin gene mutation. It received accelerated approval. Patients will take oral corticosteroids before and after receiving ELEVIDYS and will have weekly blood tests to monitor liver enzyme levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the first gene therapy for treating pediatric patients aged 4-5 years with Duchenne Muscular Dystrophy and a confirmed DMD gene mutation. VYONDYS 53, developed by Sarepta Therapeutics, is prescribed for treating DMD in patients with a specific mutation of the DMD gene that can be addressed by exon 53 skipping. This approval is under the accelerated approval pathway, based on the observed increase in dystrophin production in the skeletal muscle of patients treated with VYONDYS 53. The FDA approved VYONDYS 53 (golodirsen) in December 2019. Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical Entity (NCE) exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026. To know more about DMD treatment options, visit @ New Treatment for Duchenne Muscular Dystrophy The DMD market is crowded with so many companies including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others are running clinical trials to improve the treatment space. The DMD pipeline possesses potential drugs in mid-stage developments to be launched shortly. Some of the promising drugs in the pipeline include Pizuglanstat (TAS-205), SRP-5051, Allogeneic Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat), and others that hold the potential to create a significant positive shift in the DMD market size. Discover which therapies are expected to grab major DMD market share @ Duchenne Muscular Dystrophy Market Report Italfarmaco is developing Givinostat (ITF2357), an oral small molecule that functions as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic properties. ITF2357 increases the production of follistatin protein in muscle cells by inhibiting the HDAC enzyme, leading to increased muscle mass and prevention of muscle degeneration. This action counters the effects of myostatin, helping to alleviate the symptoms of DMD. Givinostat is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, the European Commission has designated Givinostat as an Orphan Medicinal Product for DMD treatment. TAS-205 (pizuglanstat) is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho Pharmaceutical. It is being advanced as a treatment for Duchenne muscular dystrophy (DMD), effective irrespective of the type of dystrophin gene mutation. By inhibiting HPGDS, which worsens the inflammatory response in the muscles of DMD patients, it helps control the decline in motor function. Currently, it is in Phase III of clinical trials. Discover more about drugs for DMD in development @ Duchenne Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for Duchenne muscular dystrophy are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DMD is expected to grow from USD 2.1 billion in 2023 with a significant CAGR by 2034. The growth of the DMD market is expected to be mainly driven by increased diagnosed incidence, patient awareness, and a robust clinical pipeline during the forecast period (2024–2034). DelveInsight's latest published market report titled as Duchenne Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DMD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DMD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DMD market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DMD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DMD market. Also, stay abreast of the mitigating factors to improve your market position in the DMD therapeutic space. Related Reports Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3Drug ApprovalOrphan DrugFast Track

29 May 2024

·www.fiercepharma.com

Evidence doesn't support FDA's use of surrogate markers for accelerated approvals: report

A recent study by researchers from Yale School of Medicine and Emory University shows that there is little evidence backing the FDA's use of surrogate markers to determine if a medicine is fit for an accelerated approval. In the FDA’s push to grant accelerated approvals to treatments that can help patients in need, the agency has become increasingly reliant on the use of surrogate markers (or “proxies”), which can foreshadow clinical outcomes. While it can take several years for a drugmaker to conduct a clinical trial to prove the worth of a medicine, proxies can be generated quickly and suggest that a product is ready to help cure diseases and save lives. But there is much debate—internal and external—about how much the U.S. regulator should depend on surrogate data. Adding fuel to the fire is a recent report from the Yale School of Medicine and Emory University which shows that there is little evidence backing the use of surrogate markers to determine if a medicine is fit for approval. Because a 2022 report, which limited its analysis to cancer approvals, came to a similar conclusion, the Yale and Emory researchers limited their study to non-cancer treatments that have been approved by the FDA. There was a “real paucity of evidence” that surrogates are valid markers of a good disease outcome, according to Joseph Ross, M.D., a professor of medicine and public health at Yale and the author of the study. “Even though the FDA has indicated the level of evidence needed to support use of a surrogate and feel good about its reliability, we weren’t able to find that evidence,” Ross told the Yale School of Public Health. The FDA has said that meta-analysis studies, which compile data from several different trials in a particular indication, act as a guide to determine which surrogate markers are associated with positive outcomes for patients. But Yale and Emory researchers, who analyzed 37 surrogate markers for 32 different diseases, said meta-analyses existed for only 40%. And of these, there was a lack of “high-strength evidence of associations with clinical outcomes." Once the FDA approves a drug based on biomarker data, other companies are free to pursue nods for their treatments through the same pathway. Last month, an FDA panel voted unanimously to allow a surrogate endpoint to support multiple myeloma approvals. Over the last decade, FDA experts have not been in unison on the value of surrogate markers. In 2016, for example, there was much debate on whether to approve Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 based on biomarker data. The regulator approved the treatment despite getting a thumbs down from its own reviewers and from an independent panel of experts. The disagreement was such that there were some high-profile departures from the agency. In 2020, amid a similar debate, the FDA signed off on NS Pharma’s DMD drug Viltepso based on its ability to produce the protein dystrophin. Earlier this week, the company reported that Viltepso flunked a confirmatory trial.

Drug ApprovalAccelerated Approval

28 May 2024

·www.fiercepharma.com

NS Pharma's Duchenne therapy Viltepso fails confirmatory trial

NS Pharma's Viltepso has flunked a confirmatory trial, placing its 2020 accelerated FDA approval for Duchenne muscular dystrophy in jeopardy. Since NS Pharma launched its Duchenne muscular dystrophy (DMD) drug Viltepso (viltolarsen) in 2020, sales have steadily trended upward, with the company expecting the treatment to generate 20,100 trillion Japanese yen ($128 million) this year. But the regulatory status of Viltepso may soon be in jeopardy as preliminary results from a confirmatory trial show that it has failed to reach its primary endpoint. In the study of 77 boys, while Viltepso allowed patients to stand faster after 48 weeks of treatment, those in the control group also showed increased velocity in rising to their feet. There was no significant statistical difference between the study groups. “We are currently conducting further detailed analyses and identifying factors that may have influenced the results,” NS Pharma CEO Tsugio Tanaka said in a release. “Considering the results of prior clinical studies, we have confidence that viltolarsen can be a beneficial treatment for amenable patients with Duchenne.” New Jersey-based NS Pharma—which is a subsidiary of Japan’s Nippon Shinyaku—said it is investigating the age of the participants, the treatment period and the effect of concomitant drugs including glucocorticoid therapy, which may have influenced the study. In August of 2020, the FDA granted Viltepso accelerated approval for patients with DMD amenable with exon 53 skipping. The nod was based in part on the results of a phase 2, open-label, long-term extension study of 16 participants between the ages of 4 and 10. After 205 weeks of treatment with viltolarsen, the patients showed statistically significant improvement in their time to stand compared to a historical control group of patients matched for key factors. Another factor that played into the approval was that patients in two trials showed that viltolarsen provided an increase in dystrophin levels. DMD is a genetic disorder that prevents the production of dystrophin protein, which is crucial to muscle function. Basing approvals on biomarker data has gotten the FDA in hot water before, including in the DMD field. Sarepta's FDA approvals for Vyondys 53 and sister med Exondys 51 fueled concerns for some pharma watchers because the company lacked evidence to show that the drugs could actually improve symptoms or fend off disease progression. In both cases, the FDA approved the drugs based on the drugs' ability to spark dystrophin production. The agency’s own reviewers recommended against approval of Exondys 51, and an FDA panel of independent experts narrowly voted against approval. But the agency approved it anyway, creating a dramatic scene where internal disagreement among FDA staffers led to some high-profile departures. Vyondys 53 was initially rejected, but after the company disputed the FDA's complete response letter, the agency changed its mind.

Clinical ResultDrug ApprovalAccelerated ApprovalPhase 2

100 Deals associated with Viltolarsen

External Link

KEGG	Wiki	ATC	Drug Bank
D11528	-	J05AB	DB15005

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Duchenne Muscular Dystrophy Exon 53 Skipping Mutation	JP	Nippon Shinyaku Co., Ltd.	25 Mar 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	NDA/BLA	CN	Fuji Yakuhin Co., Ltd.	10 Aug 2021
Muscular Dystrophy, Duchenne	NDA/BLA	CN	Nippon Shinyaku Co., Ltd.	10 Aug 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04060199 (RACER53, Corporate Publications) Manual	Phase 3	Muscular Dystrophy, Duchenne	77	viltolarsen	qnlpnolrde(dyggoptjjn) = there was no statistically significant difference between the viltolarsen group and the placebo group. dtzrcufvul (jfzciuwsha ) View more	Negative	27 May 2024
				Placebo
NCT04956289 (MDA2024)	Phase 2	Muscular Dystrophy, Duchenne dystrophin mutations	20	Viltolarsen 80 mg/kg/week	djcbealour(inrxrsgifr) = 19% of viltolarsen-treated participants reported a treatment-emergent adverse event; 4 events were assessed as drug-related. No serious adverse events or deaths were reported. No participants discontinued. bslmvutpry (iosinnvfom )	Positive	03 Mar 2024
NCT03167255 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	16	NS-065/NCNP-01 (NS-065/NCNP-01 40mg/kg)	drrozpocsm(hykfsutiut) = aqixjtqxwx bybkbhmpfs (tvoynpadck, kgubizbkvi - pireziofmy) View more	-	18 Nov 2022
				NS-065/NCNP-01 (NS-065/NCNP-01 80mg/kg)	drrozpocsm(hykfsutiut) = mbqlganhfl bybkbhmpfs (tvoynpadck, inejxvdsqu - ihsdeqmhqd) View more
NCT03167255 (Corporate Publications) Manual	Phase 2	Muscular Dystrophy, Duchenne Exon 53 Skipping	16	glucocorticoid treatment+Viltolarsen	kzkhewvpzq(flsrpyifwr) = bskgmydiwj bgbstdggym (iybsrzuhhd ) View more	Positive	24 Oct 2022
				glucocorticoid treatment	kzkhewvpzq(flsrpyifwr) = oocqlqqmjy bgbstdggym (iybsrzuhhd ) View more
NCT02740972 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	16	NS-065/NCNP-01 (NS-065/NCNP-01 40mg/kg)	grhklnapdv(tpwcyslytr) = piuphduxlw dakeaxkabr (bhgosxxefd, hwhodwxrnb - enqosqajzs) View more	-	12 Jul 2021
				NS-065/NCNP-01 (NS-065/NCNP-01 80mg/kg)	grhklnapdv(tpwcyslytr) = kspnxlvdqi dakeaxkabr (bhgosxxefd, zfckngliws - gjayzaejmt) View more
Corporate Publications Manual	Phase 2	Muscular Dystrophy, Duchenne Exon 53 Skipping	16	viltolarsen	buaozziocl(pzivusavru) = pghvnnpmbm rgfyddcaqa (nsfbannxlb ) View more	Positive	01 Jul 2021
				historical control	buaozziocl(pzivusavru) = mthkfhpoqe rgfyddcaqa (nsfbannxlb ) View more
NCT02740972 (Pubmed \| JAMA Neurol)	Phase 2	Muscular Dystrophies	16	Viltolarsen 40 mg/kg	peykenibkb(tbuhujrhwj) = sgrqqgwmwe zmktefrkan (gxzyqhoprt )	-	26 May 2020
				Viltolarsen 80 mg/kg	peykenibkb(tbuhujrhwj) = ujglnferlg zmktefrkan (gxzyqhoprt )
NCT02081625 (Pubmed \| Sci Transl Med) Manual	Phase 1	Muscular Dystrophy, Duchenne Exon 53 Skipping	10	NS-065/NCNP-01	hvbenryzqy(sjguldvgiu) = pauzasbgpo ejeslcovef (ijhaidnckl ) View more	Positive	18 Apr 2018

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