Do We Get Samples of the Report?

Yes, you will receive a sample copy of the market research report from the sales team when you put a query to us. The sample report is free and offers a brief view of the report and information on data available before you purchase the full-fledged report. Also, we will provide a demo sample for any oncoming study.

How to Buy a Market Report?

Select the report you wish to purchase and proceed to buy it by making an online payment. We use a secured gateway for payment transfers and do not share your financial information with any unconcerned third party.

In Which Format Will I Get the Report?

The report will be delivered in spreadsheet and PDF format. In some cases, PPT formats could be available as well.

In How Much Time I will Get the Report After Purchase?

After confirmation of the payment, you will receive the electronic format of the report at your registered email address in (mention the timeline for the delivery). If you buy a report over a holiday or weekend, we will process the request the next working day.

How Can I Get the Report Customized?

Market studies and reports already contain important data and are ready for distribution to the purchaser. However, if there is any particular query, addition, or customization you require, then contact us on our email id or phone. We will look to accommodate your request and see what can be done at the earliest. The research team will design the report accordingly and discuss the solutions beforehand with you.

How Much Will It Cost to Get a Report Customized?

The price of the customized report will be conveyed to you by our team after we agree on the deliverable. A delivery timeline will be set so that you get the report within the expected date. Any requests for customizations after selling the report will be handled by a separate department and the prices for the same mentioned upfront.

Can I Buy Only a Particular Segment of the Study?

While usually such a request is not accommodated as any market research report is wholesome and the cost of the report is for overall contents, in special cases, we may be able to sell select segments of the report based on your requirements.

Is There Any Available Discount?

Our team will love to support academicians, start-ups, students, and others with consulting and research. Thus, our pricing plan is based on keeping these interests in mind and is already affordable. However, we may have discounts available for particular customer types and reports. So, contact us for more information.

Which Services Are Complimentary Before Purchasing the Report?

We have consumer-centric reports and offer several facilities before selling. You will get a free review and sample report, talk to our customer care team for any additions to be done and meet special requirements, and interact with our analysts for deeper understanding.

Will I Receive a Bill or Invoice for the Report Purchased?

Yes, you will get an invoice for the purchase you made. The invoice will be sent to you electronically on your registered email id.

What If I am Unable to Find the Report in the Catalog?

We have a vast database of market reports and studies and there are a few which may not have been uploaded yet. If you are unable to find the one you want, then get in touch with our sales team by phone or email and we will connect with you for assistance.

How Often Are the Existing Studies Updated?

Not every study is the same. Thus, the update for reports will depend on the ongoing research and changes if any to be incorporated in the document or not.

What If the Report Does Not Contain the Information I Want?

The market research reports are constructed keeping in mind the industry trends, nature of the study, and required inputs. But if you want something more than what is stated in the report, we can help you out with further research if possible. Contact us right away, and we will do the needful to aid you.

Is the Payment Refundable?

Once the payment is done, it is non-refundable because as per the standard policy, the utility of the reports is based on knowledge sharing, and thus, a refund is not possible to make after we have issued you obtain the study. To avoid the hassle, we have a detailed view of the report and its ideas through a sample report.

What If the Payment Fails?

If the payment fails then be assured that you shall receive the money back to the source payment mode from the payment gateway. Aarkstore is not responsible for any loss of payment in the process.

Can I Avail the Report of a Particular Country/Region?

Yes, we can provide a report or segment related to a particular region or geography if we have that feature available for the document.

Do You Offer Support After Purchase?

Yes, we are here to solve any queries you may have for the report from our analyst till a given timeframe. To understand more about this, get in touch with us.

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2017

DESCRIPTION

TABLE OF CONTENTS

RELATED REPORTS

SAMPLE REQUEST

REVIEWS

DESCRIPTION

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2017

Summary

Global Markets Directs latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I - Pipeline Review, H2 2017, provides an overview of the Mucopolysaccharidosis I (Metabolic Disorders) pipeline landscape.

MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

Report Highlights

Global Markets Directs Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Mucopolysaccharidosis I (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed, Preclinical and Discovery stages are 5, 1, 6 and 2 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

Mucopolysaccharidosis I (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Directs proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Mucopolysaccharidosis I (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis I (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis I (Metabolic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Mucopolysaccharidosis I (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Mucopolysaccharidosis I (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and Scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

TABLE OF CONTENTS

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
AngioChem Inc
ArmaGen Inc
CRISPR Therapeutics
Immusoft Corp
JCR Pharmaceuticals Co Ltd
OPKO Health Inc
PTC Therapeutics Inc
RegenxBio Inc
Sangamo Therapeutics Inc
Sevion Therapeutics Inc (Inactive)
Tamid Bio Inc
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
AGT-181 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Antisense Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ataluren - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ELX-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-171 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pentosan polysulfate sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RGX-111 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-318 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Hurler Sydrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Tamid-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
X-372 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
Featured News & Press Releases
Dec 08, 2017: Sangamo Announces EMA Recommendation of Orphan Medicinal Product Designation for Investigational Genome Editing Treatment for Mucopolysaccharidosis Type I
Nov 30, 2017: ArmaGens AGT-181 Granted Fast Track Designation for the Treatment of Hurler Syndrome (MPS I)
Oct 26, 2017: JCR to Initiate Development of JR-171, a New Drug Candidate for Hurler Syndrome Using J-Brain Cargo
Aug 08, 2017: REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I
Jul 13, 2017: Sangamo Receives Fast Track Designation From The FDA For SB-318 In Vivo Genome Editing Product Candidate For The Treatment Of MPS I
Feb 27, 2017: Sangamo Therapeutics Receives Rare Pediatric Disease Designation From FDA For SB-318 In Vivo Genome Editing Therapeutic For MPS I
Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
Feb 08, 2017: Sangamo Therapeutics Announces Data on SB-318 at The 13th Annual WORLDSymposium Meeting
Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
Jan 11, 2017: Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
May 09, 2016: Sangamo BioSciences Presents Recent Developments From Research And ZFP Therapeutic Programs In Multiple Presentations At Annual Meeting of the American Society of Gene and Cell Therapy
Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products under Development by Universities/Institutes, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Universities/Institutes, H2 2017
Number of Products by Stage and Target, H2 2017
Number of Products by Stage and Mechanism of Action, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sevion Therapeutics Inc (Inactive), H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Tamid Bio Inc, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H2 2017
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H2 2017

List Of Figures

Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products under Development by Universities/Institutes, H2 2017
Number of Products by Stage and Targets, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017

RELATED REPORTS

Fill The Form For Sample Request

Full Name*

Business Email *

E-mail

Country *

Contact No *

Alternate No

Note : * Kindly provide us with your company id and get the sample reports at the earliest.

REVIEWS

There is no Reviews available

Search and Get the Latest Market Research Reports

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H2 2017

Mucopolysaccharidosis VI (MPS VI) (Maroteaux-Lamy Syndrome ) (Genetic Disorders) - Drugs in Development, 2021

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Disease - Global Clinical Trials Review, H2, 2020

Mucopolysaccharidosis VI (MPS VI) (Maroteaux-Lamy Syndrome ) (Genetic Disorders) - Drugs in Development, 2021

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Disease - Global Clinical Trials Review, H2, 2020

FAQs