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Myotonic Dystrophy Pipeline Appears Robust With 25+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

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New York, USA, Feb. 15, 2023 (GLOBE NEWSWIRE) — Myotonic Dystrophy Pipeline Appears Robust With 25+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood.The current therapeutic developments at RNA or DNA levels should be applicable in principle to the CNS. Therapeutic molecular tools to eliminate toxic RNAs, release the sequestrated mediators, or to remove expanded repeats in the DMPK gene should also be efficient in brain cells. The most recent strategies to counteract MDs concern gene therapy and the repurposing of drugs. Maximizing health and functional independence is the goal of supportive management. This is geared towards monitoring and treating all the medical issues associated with DM.The rapid technological advancements in cellular and molecular biology in genomics research have contributed greatly to the growing gene therapy market in recent years.

DelveInsight’s Myotonic Dystrophy Pipeline Insight – 2023 report provides comprehensive global coverage of available, marketed, and pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain.

Key Takeaways from the Myotonic Dystrophy Pipeline Report

  • DelveInsight’s myotonic dystrophy pipeline report depicts a robust space with 25+ active players working to develop 28+ pipeline therapies for myotonic dystrophy treatment. 
  • Key myotonic dystrophy companies such as Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others are evaluating new drugs for myotonic dystrophy to improve the treatment landscape.
  • Promising myotonic dystrophy pipeline therapies in various stages of development include Mexiletine, Tideglusib, Pitolisant, AOC 1001, DYNE-101, NXEN04, Research programme: neuromuscular gene therapies, ENTR-701, ATX-01, NT-0200, NT 0231 F, ENZ-001, ENZ003, AT 466, Dyne-102, PGN EDODM1, Research Programme: Type1 Muscular Dystrophy, Research programme: myotonic dystrophy therapeutics, and others.
  • In August 2022, the US granted Arthex Biotech’s lead investigational product ATX-01 Orphan Drug Designation (ODD). Food and Drug Administration (FDA). ATX-01 is under development for the treatment of Myotonic Dystrophy Type 1 (DM1), a serious, debilitating, and life-threatening disease with no known cure or currently available drug treatment.
  • In July 2022, Dyne Therapeutics, Inc. announced that the New Zealand Medicines and Medical Devices Safety Authority cleared its clinical trial application to initiate its Phase I/II multiple ascending doses (MAD) clinical trials of DYNE-101 in patients with myotonic dystrophy type 1 (DM1).
  • In May 2022, AMO Pharma Limited announced that current investors had made additional investments in the Company in response to progress in AMO Pharma’s potentially pivotal REACH-CDM study, a double-blind, placebo-controlled, randomized clinical trial to assess the efficacy and safety of AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy (CDM1).

Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report

The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape.

Myotonic Dystrophy Overview

Steinert, Batten, and Gibb identified myotonic dystrophy (DM) as a rare progressive disorder that is characterized by weakness, especially in muscles. Alterations in the central nervous system also characterize it. There are two major types of myotonic dystrophy—Steinert disease or myotonic dystrophy type 1 (DM1) and a milder form, DM2 or proximal myotonic myopathy. The myotonic dystrophy symptoms vary from person to person and even among the subtypes of myotonic dystrophy despite the overlap. These constellations of myotonic dystrophy symptoms make myotonic dystrophy a multifaceted and complex disorder. 

A thorough clinical evaluation, a detailed patient and family history, and identifying characteristic physical findings may lead to a diagnosis of myotonic dystrophy. Several laboratory tests, including blood tests, electromyography (EMG), magnetic resonance imaging (MRI), muscle biopsy, and genetic testing, can help to clarify the clinical myotonic dystrophy diagnosis. Though various diagnostic options exist, the definitive test for myotonic dystrophy is a genetic test. 

Currently, no approved treatment provides a permanent cure for myotonic dystrophy, but researchers are looking into ways to help people with these disorders. The current myotonic dystrophy treatment focuses on each individual’s specific symptoms.

Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment

A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report:

Drugs Company Phase  MoA RoA
Mexiletine Lupin Phase III Sodium channel antagonists Oral
AMO-02 AMO Pharma Phase II/III Glycogen synthase kinase 3 beta inhibitors Oral
Pitolisant Harmony Biosciences Phase II Histamine H3 receptor antagonists; Histamine H3 receptor inverse agonists Oral
AOC 1001 Avidity Biosciences Phase II Myotonin protein kinase modulators; RNA interference Intravenous
DYNE-101 Dyne Therapeutics Phase I/II Myotonin protein kinase expression inhibitors; RNA interference Intravenous
ENTR-701 Entrada Therapeutics Preclinical RNA inhibitors Parenteral

Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials

Myotonic Dystrophy Therapeutics Assessment

The myotonic dystrophy pipeline report proffers an integral view of myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Myotonic Dystrophy Pipeline Report 

  • Coverage: Global 
  • Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
  • Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Therapeutics Assessment By Route of Administration: Intravenous, Oral, Subcutaneous
  • Therapeutics Assessment By Molecule Type: Small Molecule, Antidepressants
  • Therapeutics Assessment By Mechanism of Action: Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, Myotonin protein kinase expression inhibitors, RNA interference, Myotonin protein kinase modulators, RNA inhibitors
  • Key Myotonic Dystrophy Companies: Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals,  and others.
  • Key Myotonic Dystrophy Pipeline Therapies: Mexiletine, Tideglusib, Pitolisant, AOC 1001, DYNE-101, NXEN04, Research programme: neuromuscular gene therapies, ENTR-701, ATX-01, NT-0200, NT 0231 F, ENZ-001, ENZ003, AT 466, Dyne-102, PGN EDODM1, Research Programme: Type1 Muscular Dystrophy, Research programme: myotonic dystrophy therapeutics, and others.

Dive deep into rich insights for new drugs for myotonic dystrophy treatment; visit @ Myotonic Dystrophy Medications

Table of Contents

1. Myotonic Dystrophy Pipeline Report Introduction
2. Myotonic Dystrophy Pipeline Report Executive Summary
3. Myotonic Dystrophy Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Myotonic Dystrophy Clinical Trial Therapeutics
6. Myotonic Dystrophy Pipeline: Late Stage Products (Pre-registration)
7. Myotonic Dystrophy Pipeline: Late Stage Products (Phase III)
8. Myotonic Dystrophy Pipeline: Mid Stage Products (Phase II)
9. Myotonic Dystrophy Pipeline: Early Stage Products (Phase I)
10. Myotonic Dystrophy Pipeline Therapeutics Assessment
11. Inactive Products in the Myotonic Dystrophy Pipeline
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products in the Myotonic Dystrophy Pipeline
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. 

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Artificial Intelligence

nodeQ Launches PQtunnelTM: The Leading-Edge Cybersecurity Solution for Quantum-Safe Communication

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The latest application developed by nodeQ simplifies the migration to Post-Quantum Cryptography for both SMEs and large enterprises
YORK, England, May 7, 2024 /PRNewswire/ — The advent of Quantum Computing poses a significant threat to existing public-key cryptosystems. A large quantum computer can break algorithms like RSA or ECDH, which form the backbone of current internet security. Already today, confidential data is in danger: It can be harvested now to be decrypted later when a quantum computer can run Shor’s factorization quantum algorithm on a sufficiently large number of qubits.

This vulnerability exposes a large number of applications to potential security breaches, threatening our trillion-dollar digital economy. Multiple sectors are under threat: Financial Institutions, government entities, telecommunications companies, cloud providers, the healthcare industry, the defense sector, universities and research centers, and virtually any IT-based enterprise that relies on encrypted data.
Companies must transition IT infrastructures to quantum-resistant solutions, such as Post-Quantum Cryptography (PQC). In the US the transition to PQC is set to start in 2024, as a result of Biden’s 2022 Computing Cybersecurity Preparedness Act and the end of NIST standardization.
nodeQ has developed PQtunnel™, a groundbreaking tool designed to assist businesses – ranging from SMEs to large enterprises – in transitioning their end-to-end (E2E) secure communication to PQC. This innovative software application is available in two variants: PQtunnel™ TLS and PQtunnel™ SSH, meeting diverse business requirements.
PQtunnel™ TLS is an enterprise-oriented tunneling application that leverages the Transport Layer Security (TLS) protocol to secure traffic between a client (or a proxy) and the target application. This solution has been thoroughly tested on various enterprise-grade applications, such as GitLab EE, demonstrating its effectiveness and reliability.
On the other hand, PQtunnel™ SSH offers a comprehensive Quantum-Safe SSH server, alongside a client equipped with a user-friendly interface. This software suite is designed for secure shell operations, ensuring the highest level of security and ease of use.
Both PQtunnel™ variants utilize a wide range of PQC algorithms, including the most recent ones under standardization by NIST, and hybrid algorithms. This approach combines the stability of current cryptographic methods with the quantum-safeness of new cryptographic suites, offering unmatched security against emerging cyber threats.
Developed as cloud-native applications, both variants of PQtunnel™ are designed to facilitate easy distribution and provide flexibility in a wide range of scenarios. Secure Post-Quantum tunneling to your GitLab server, quantum-safe data transfer, secure cloud IT management, and PQC benchmarking are just a few of the use cases where PQtunnel™ can be applied, showcasing its versatility and significance for today’s cybersecurity landscape.
Ignazio Pedone, the cybersecurity lead at nodeQ, emphasized the significance of their latest security product, stating, “This security product represents an important step towards a more effective and robust application of quantum-safe cryptography for enterprises.”
Various early customers have already adopted PQtunnel™ to test their readiness to migrate to quantum-safe cryptography. PQC is going to play an important role for telecom operators.
“At Fibraweb, PQtunnel demonstrated our readiness for quantum-security migration,” says Walid Bounassif, CEO of Fibraweb (an Italian telco). “The software created quantum-resistant connections between external clients and our internal servers, showing that post-quantum encrypted communication can efficiently be implemented for remote IT administration and secure file transfer.”
PQC is essential for medium to large enterprises, including educational institutions like universities, where maintaining data confidentiality at various levels is crucial. At the University of York (UK), PQtunnel™ has been deployed for the quantum-safe transfer of documents. This pioneering demonstration, the first of its kind in the world within the education sector, actively involved staff and students from the Department of Computer Science.
“PQtunnel has effectively established high-speed, quantum-resistant connections both within and beyond the campus perimeter,” says Prof Paul Cairns (Head of the Computer Science Department at the University of York, UK). “This technology has clearly demonstrated its capacity for facilitating quantum-secure data transfers, affirming its readiness to enhance our university’s digital infrastructure.”
For additional information about PQtunnel™, nodeQ, and their range of products, the reader can visit their website at www.nodequantum.com.
 Key Features
Crypto-Agility: implements all NIST PQC algorithms to counteract the quantum threat.Cross-Platform: available for the most adopted operating systems and hardware architectures.Easy Setup: no need for long or complex installation procedures to get started.On-the-Fly Benchmarking and Optimization: can benchmark and optimize high-level security protocols against all the available combinations of PQC algorithms for key exchange and digital signature.Target Customers
Financial InstitutionsTelecommunications CompaniesUniversities and Research CentersCloud ProvidersHealthcare IndustryGovernment EntitiesDefense SectorManufacturing CompaniesSMEs handling confidential dataAbout nodeQ
At nodeQ, we are pioneering the future of computer networks, leveraging our deep expertise in quantum communication, artificial intelligence, and software-defined networking. Our primary objective is to transition computer networks to quantum security, placing a critical emphasis on maintaining speed, performance, and usability at the heart of this transformation.
Contacts
Stefano [email protected]
Request a demo or a brochure at [email protected] or visit www.nodequantum.com.
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Simplilearn and Purdue University Online Unveil Cutting-Edge Generative AI Program for Business Transformation

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The program provides over 50 hours of live online sessions with industry experts, exploring GenAI tools like ChatGPT, Gemini and Amazon CodeWhisperSuitable for candidates with preferably over 5 years of work experience, proficiency in English and an eagerness to leverage Gen AI to solve real business problemsThe program offers the applications of Generative AI in various business functions like software engineering, sales and marketing, customer service, product research, and more.SAN FRANCISCO, May 7, 2024 /PRNewswire/ — Simplilearn, the world’s leading online bootcamp provider, has strengthened its partnership with Purdue University, renowned for its unwavering commitment to innovation, research, and education. Together, they proudly announced the launch of the  Generative AI for Business Transformation program. As the global demand for Generative AI skyrockets, with projections* soaring to $667.9 billion market by 2030, boasting a staggering annual growth rate of 24.4%, this program emerges as a pivotal force in the transformative journey into the Generative AI domain.

The program offers a comprehensive understanding of Generative AI and its applications across business functions such as sales, marketing, software engineering, customer service, data analytics, and product research & development. It empowers learners with the skills to automate tasks, predict trends, generate code, and personalize content using Generative AI and navigate its ethical considerations. From automated copywriting to predictive analytics, the program helps learners foster responsible and effective use of GenAI across diverse domains.
Learners can expect a dynamic learning experience with over 50 hours of live online classes, exposure to prominent Generative AI tools, and masterclasses conducted by the Purdue faculty and staff. Professionals will master the essentials of Generative AI, including LLMs, prompt engineering, ChatGPT, its applications and benefits to drive business outcomes.
Upon successfully completing the program, learners will receive a certificate jointly issued by Purdue University Online and Simplilearn and access to Purdue’s Alumni Association membership for global recognition.
The program is suitable for professionals with over 5 years of work experience, proficiency in English and an eagerness to leverage Generative AI to solve real business problems. The program is designed for a diverse range of professionals, including business Leaders, C-suite executives, mid-senior level managers, functional heads, consultants, technology professionals, data professionals and entrepreneurs.
Speaking about the program, Mr. Anand Narayanan, Chief Product Officer of Simplilearn, said,”Our collaboration with Purdue University Online brings together the excellence of the world’s leading online bootcamp provider and leading academic institution known for its commitment to innovation, research, education, and expertise in the AI and ML fields. This program will empower business leaders, mid-senior level managers, functional heads, and technology professionals with the skills to stay at the forefront of industry advancements. With Gen AI at its core, learners will be equipped to navigate in today’s dynamic AI landscape, fostering responsible innovation, and business transformation ideas.”
Dimitrios Peroulis, Senior Vice President for Purdue University Online, said, “As a beacon of academic excellence, Purdue University Online is excited to partner with Simplilearn in delivering a transformative learning experience through the Generative AI for Business Transformation program. This collaboration reflects our commitment to providing high-quality education and preparing professionals for the challenges and opportunities presented by emerging technologies like Generative AI.”
About Purdue University
Purdue University is a public research institution demonstrating excellence at scale. Ranked among top 10 public universities and with two colleges in the top four in the United States, Purdue discovers and disseminates knowledge with a quality and at a scale second to none. More than 105,000 students study at Purdue across modalities and locations, including nearly 50,000 in person on the West Lafayette campus. Committed to affordability and accessibility, Purdue’s main campus has frozen tuition 13 years in a row. See how Purdue never stops in the persistent pursuit of the next giant leap — including its first comprehensive urban campus in Indianapolis, the new Mitchell E. Daniels, Jr. School of Business, and Purdue Computes — at:
https://www.purdue.edu/president/strategic-initiatives 
About Simplilearn
Founded in 2010 and based in San Francisco, California, and Bangalore, India, Simplilearn, a Blackstone portfolio company, is the world’s #1 online Bootcamp for digital economy skills training. Simplilearn offers individuals and businesses worldwide access to world-class work-ready training. The Bootcamps are designed and delivered with world-renowned universities, top corporations, and leading industry bodies via live online classes featuring top industry practitioners, sought-after trainers, and global leaders. From college students and early career professionals to managers, executives, small businesses, and big corporations, Simplilearn’s role-based, skill-focused, industry-recognized, and globally relevant training programs are ideal upskilling solutions for diverse career or business goals.
Simplilearn conducts more than 3,000 live classes monthly, with an average of 80,000 learners who spend more than 500,000 hours on the platform. Its programs allow learners to upskill and get certified in popular domains. 
For more information, please visit www.simplilearn.com/
*Fortune Business Insights.
 
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NetSuite Awarded Frost & Sullivan’s 2024 Global Company of the Year Award for Leading Innovation in Cloud Business Management Solutions

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NetSuite revolutionizes cloud enterprise resource planning (ERP) with its all-in-one cloud business suite that helps drive efficiency and visibility for organizations worldwide
SAN ANTONIO, May 7, 2024 /PRNewswire/ — Frost & Sullivan recently researched the cloud ERP market and, based on its findings, recognizes Oracle NetSuite with the Global Company of the Year Award. NetSuite is an integrated cloud business system that helps businesses simplify core processes, improve productivity, expand insights, and grow more efficiently.

View the full multi-media landing page: https://best-practices.frost.com/oracle-netsuite/
NetSuite’s leadership in the cloud ERP space is evident with more than 37,000 customers across diverse sectors, including education, energy, finance, healthcare, manufacturing, and retail. Additionally, the company adopts a pioneering approach to embedding artificial intelligence (AI), which makes it stand out in the market. Leveraging Oracle Cloud Infrastructure’s advanced supercluster technology, NetSuite provides powerful AI capabilities that deliver actionable insights and increase the speed and accuracy of business processes. By embedding generative AI capabilities across the suite, NetSuite empowers global organizations to maximize productivity and make informed decisions based on real-time insights.
With NetSuite, upgrades are seamlessly delivered so customers are always using the latest version of its software, which improves access to cutting-edge features. Furthermore, the company’s extensive partner and professional services ecosystem significantly enhance the customer experience, enabling businesses to optimize the value of their NetSuite investment.
“Opportunities for AI include automation (data entry, financial analysis, and inventory management), actionable insights (better decision-making using real-time insights and predictions about customers and competitors), human-like interaction (integrating machine learning (ML) and natural language processing), and anomaly detection (enhanced security and detection of unexpected patterns). NetSuite currently has all these elements in its product suite. AI is embedded in business processes within NetSuite and also available in the form of assistants and advisors,” said Sankara Narayanan, Industry Director at Frost & Sullivan.
To address its customers’ growing curiosity about AI and its business implications, NetSuite leverages its Learning Lab events to provide customers and end users with hands-on training and expert guidance on how to effectively integrate AI and automation across operations. With this unique approach, NetSuite guarantees that customers have the knowledge and skills to optimally leverage AI capabilities within their businesses and connect with like-minded innovators in various cities across the U.S. and Canada.
NetSuite’s continuous focus on innovation and customers has made it stand out in the cloud ERP market, and it continues to deliver more features than most of its competitors, whose solutions are less capable than those of NetSuite’s system.
Each year, Frost & Sullivan presents a Company of the Year award to the organization that demonstrates excellence in terms of growth strategy and implementation in its field. The award recognizes a high degree of innovation with products and technologies, and the resulting leadership in terms of customer value and market penetration.
Frost & Sullivan Best Practices awards recognize companies in various regional and global markets for demonstrating outstanding achievement and superior performance in leadership, technological innovation, customer service, and strategic product development. Industry analysts compare market participants and measure performance through in-depth interviews, analyses, and extensive secondary research to identify best practices in the industry.
About Frost & SullivanFor six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders, and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models, and companies to action, resulting in a continuous flow of growth opportunities to drive future success. Contact us: Start the discussion.
Contact:Lindsey WhitakerP: +1 (210) 477-8457E: [email protected] 
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