Scleroderma, Localized

May 31, 2023 -- Researchers at the National Institutes of Health and their colleagues have identified genomic variants that cause a rare and severe inflammatory skin disorder, known as disabling pansclerotic morphea, and have found a potential treatment. Scientists discovered that people with the disorder have an overactive version of a protein called STAT4, which regulates inflammation and wound healing. The work also identified a drug that targets an important feedback loop controlled by the STAT4 protein and significantly improves symptoms in these patients. The results were published in the New England Journal of Medicine . The study was led by researchers at the National Human Genome Research Institute (NHGRI), part of NIH, in collaboration with researchers from the University of California, San Diego (UCSD) and the University of Pittsburgh. Researchers from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute of Allergy and Infectious Diseases, both part of NIH, also participated in the study. Only a handful of patients have been diagnosed with disabling pansclerotic morphea, a disorder first described in the medical literature around 100 years ago. The disorder causes severe skin lesions and poor wound healing, leading to deep scarring of all layers of the skin and muscles. The muscles eventually harden and break down while the joints stiffen, leading to reduced mobility. Because the disorder is so rare, its genetic cause had not been identified until now. “Researchers previously thought that this disorder was caused by the immune system attacking the skin,” said Sarah Blackstone, a predoctoral fellow within NHGRI's Inflammatory Disease Section, a medical student at the University of South Dakota and co-first author of the study. “However, we found that this is an oversimplification, and that both skin and the immune system play an active role in disabling pansclerotic morphea.” The researchers used genome sequencing to study four individuals with disabling pansclerotic morphea and found that all four have genomic variants in the STAT4 gene. The STAT4 gene encodes a type of protein that helps turn genes on and off, known as a transcription factor. The STAT4 protein not only plays a role in fighting infections but also controls important aspects of wound-healing in the skin. The scientists found that the STAT4 genomic variants result in an overactive STAT4 protein in these four patients, creating a positive feedback loop of inflammation and impaired wound-healing that worsens over time. To stop this harmful feedback loop, they targeted another protein in the inflammatory pathway that interacts with the STAT4 molecule and is called Janus kinase, also known as JAK. When the researchers treated the patients with a JAK-inhibiting drug called ruxolitinib, the patients’ rashes and ulcers dramatically improved. “So far, there has not been a standard treatment for this disorder because it’s so rare and not well-understood. However, our study gives an important new treatment option for these patients,” said Blackstone. Existing treatments for disabling pansclerotic morphea are designed to halt the progression of the disorder, but previous therapies have been mostly ineffective, often with severe side effects. People with the disorder typically don’t live more than 10 years after their diagnosis. The study suggests that ruxolitinib could be an effective treatment for patients with this disorder. Ruxolitinib is part of a broader class of drugs called JAK inhibitors, which are commonly used to treat arthritis, eczema, ulcerative colitis and other chronic inflammatory diseases. “The findings of this study open doors for JAK inhibitors to be a potential treatment for other inflammatory skin disorders or disorders related to tissue scarring, whether it is scarring of the lungs, liver or bone marrow,” said Dan Kastner, M.D., Ph.D., an NIH distinguished investigator, head of NHGRI’s Inflammatory Disease Section and a senior author of the paper. “We hope to continue studying other molecules in this pathway and how they are altered in patients with disabling pansclerotic morphea and related conditions to find clues to understanding a broader array of more common diseases,” said Lori Broderick, M.D., Ph.D., a senior author of the paper and an associate professor at UCSD. The National Human Genome Research Institute (NHGRI) is one of the 27 institutes and centers at the NIH, an agency of the Department of Health and Human Services. The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. Additional information about NHGRI can be found at: https://www.genome.gov. About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov . NIH…Turning Discovery Into Health ® References Baghdassarian et al (2023). Variant STAT4 and Response to Ruxolitinib in an Autoinflammatory Syndrome. New England Journal of Medicine. DOI: 10.1056/NEJMoa2202318 Source: NIH Posted: May 2023

NEW YORK, June 23, 2022 /PRNewswire/ -- As per Zion Market Research study, The global scleroderma diagnostics and therapeutics market was approximately USD 2232.5 million in 2021 and is anticipated to reach nearly USD 4127.6 million by 2028, with a compound annual growth rate (CAGR) of about 9.2 percent over the forecast period. Key Industry Insights & Finding of the Scleroderma Diagnostics and Therapeutics Market Reports: As per the analysis shared by our research analyst, the Scleroderma Diagnostics and Therapeutics Market is expected to grow annually at a CAGR of around 9.2 % (2022-2028). Through the primary research, it was established that the Scleroderma Diagnostics and Therapeutics Market was valued approximately USD 2232.5 Million in 2021 and is projected to reach to roughly USD 4127.6 Million by 2028. Major factors such as favorable reimbursement policies, heavy investment in R&D and drug development, and well-established healthcare infrastructure are boosting the growth of the market in this region. The growing prevalence of the disease and technological advancement in the field of medical devices are also propelling the growth of the market in this region. Europe is anticipated to hold the second-most position in the market owing to an increase in initiatives and proactive awareness regarding the diagnostics for scleroderma. Zion Market Research published the latest report titled as "Scleroderma Diagnostics And Therapeutics Market By Diagnostic Test Type (Blood Tests, Imaging Techniques, Skin Biopsy, Pulmonary Function Tests, And Electrocardiogram & Echocardiogram), By Indication (Systemic Scleroderma And Localized Scleroderma), By Drug Class (Prostacyclin Analogues, Endothelin Receptor Antagonists, Phosphodiesterase 5 Inhibitors – PHA, Immunosuppressors, Analgesics, Calcium Channel Blockers, And Others), And By Region – Global And Regional Industry Overview, Market Intelligence, Comprehensive Analysis, Historical Data, And Forecasts 2022 – 2028." into their research database. Scleroderma Diagnostics and Therapeutics Market : Overview Scleroderma is an autoimmune disease wherein healthy tissue is substituted with thick, dense, fibrous tissue. It is a chronic, albeit rare, condition. The immune system normally aids in the body's defense against sickness and infection. The immune system in scleroderma patients causes other cells to create excessive collagen. This excess collagen builds up in the organs and skin, causing hardness and thickness similar to the process of scarring. While scleroderma has no cure, therapy can help to alleviate symptoms, reduce progression, and enhance the quality of life. Scleroderma can impact several other body parts, such as the gastrointestinal system, joints, muscles, blood vessels, kidneys, and lungs, in addition to the skin. Scleroderma can be life-threatening in its most severe forms. Get a Free Sample Report with All Related Graphs & Charts (with COVID 19 Impact Analysis): Our Free Sample Report Includes: 2022 Updated Report Introduction, Overview, and In-depth industry analysis COVID-19 Pandemic Outbreak Impact Analysis Included 180 + Pages Research Report (Inclusion of Updated Research) Provide Chapter-wise guidance on Request 2022 Updated Regional Analysis with Graphical Representation of Size, Share & Trends Includes Updated List of tables & figures Updated Report Includes Top Market Players with their Business Strategy, Sales Volume, and Revenue Analysis Zion Market Research methodology Industry Dynamics: Scleroderma Diagnostics and Therapeutics Market : Growth Drivers Growing prevalence of scleroderma is fostering the growth of the market. In developed as well as developing countries, the prevalence of scleroderma is growing at a rapid rate. This condition affects between 75,000 to 100,000 persons in the United States, the majority of whom are females between the ages of 30 to 50. Twins and family members of people with scleroderma or even other autoimmune connective tissue illnesses like lupus are at an increased risk of developing the condition. Scleroderma can affect children, although the condition affects them differently than it does adults. Such a huge burden of disease has led to increase in awareness and need for diagnosis of the disease. This is ultimately driving the market growth. In addition to this, favorable reimbursement policies, well-developed health infrastructure, and growing necessity for developed diagnostic techniques are some of the key factors that are boosting the growth of the market for scleroderma diagnostics and therapeutics. Scleroderma Diagnostics and Therapeutics Market : Restraints High cost associated with scleroderma diagnostic and treatment is likely to hamper the market growth. The multiorgan nature of systemic sclerosis necessitates long-term monitoring and a variety of approaches to patient management. As a result, it's no surprise that systemic sclerosis is one of the most expensive rheumatic illnesses, with patients using more healthcare dollars per year than their age and sex-matched colleagues with inflammatory myopathies, psoriatic arthritis, and/or RA. Additionally, some of the direct costs for patients with systemic sclerosis include acute-care hospitalization, assistive devices, medications, diagnostic tests as well as visits to healthcare professionals. Thus, the financial cost of systemic sclerosis is substantial, and disease & health conditions have a significant influence on the economic burden thereby impeding the growth of the global scleroderma diagnostics and therapeutics market. In addition to this, factors such as low penetration of the market in underdeveloped countries, adverse effects associated with the drugs, and low spending power in rural areas are also hampering the market growth. Directly Purchase a Copy of the Report @ Global Scleroderma Diagnostics and Therapeutics Market : Opportunities Growing focus on development of innovative methods is estimated to drive the market during the forecast period. Systemic sclerosis is a multifaceted autoimmune disease with a wide range of symptoms and degrees of internal organ damage. All consequences have a substantial influence on the patient's quality of life as well as morbidity and death rates. As a result, there is a growing interest in novel ways of assessing, diagnosing, and monitoring organ involvement. In addition to this, there is an increase in investment in R&D activities by major players for the development of new and effective treatments. Major players are also focusing on the completion of clinical trials for the drugs and looking forward to the approval of the drug for the treatment of scleroderma. All such factors coupled with technological advancements including the implementation of artificial intelligence and internet of things in the field of medical devices are anticipated to generate numerous opportunities for the growth of the global scleroderma diagnostics and therapeutics market during the forecast period. Global Scleroderma Diagnostics and Therapeutics Market : Challenges Strict regulations for the approval of drugs and diagnostic devices for scleroderma may act as a challenge to the market growth. By far there are very less approved drugs for the treatment of scleroderma. Scleroderma is a rare disease and its diagnosis is also challenging. The vague clinical presentation of scleroderma might make diagnosis difficult, resulting in a delayed diagnosis. This also interferes in the development of drugs that can be effectively used to treat the disease. The lack of sufficient data on the efficacy and safety of the drugs leads to the failure of clinical trials. The regulatory bodies across different countries have imposed strict regulations for the approval of drugs and diagnostic devices in order to have appropriate diagnosis and treatment of the disease. This may act as a major challenge to the growth of the market. Global Scleroderma Diagnostics and Therapeutics Market : Segmentation The global scleroderma diagnostic and therapeutics market is categorized based on diagnostic test type, indication, drug class, and region. The diagnostic test type segment comprises blood tests, imaging techniques, skin biopsy, pulmonary function tests, and electrocardiogram & echocardiogram. Based on the indication, the global market is bifurcated into systemic scleroderma and localized scleroderma. The drug class segment is classified into prostacyclin analogues, endothelin receptor antagonists, phosphodiesterase 5 inhibitors – PHA, immunosuppressors, analgesics, calcium channel blockers, and others. Get More Insight before [email protected] : List of Key Players of Scleroderma Diagnostics and Therapeutics Market : Bayer AG; Boehringer Ingelheim International GmbH arGentis Pharmaceuticals LLC Celgene Corp. Bristol-Myers Squibb Company F. Hoffman La-Roche Ltd. Corbus Pharmaceuticals Holdings Seattle Genetics Inc. Kadmon Holdings Inc. Emerald Health Pharmaceuticals Prometic Life Sciences Inc. Key questions answered in this report: What are the growth rate forecast and market size for Scleroderma Diagnostics and Therapeutics Market ? What are the key driving factors propelling the Scleroderma Diagnostics and Therapeutics Market forward? What are the most important companies in the Scleroderma Diagnostics and Therapeutics Market Industry? What segments does the Scleroderma Diagnostics and Therapeutics Market cover? How can I receive a free copy of the Scleroderma Diagnostics and Therapeutics Market sample report and company profiles? Report Scope: Free Brochure: Recent Developments In March 2021, Roche received approval for Actemra®/RoActemra® (tocilizumab) subcutaneous injection from the US Food and Drug Administration (FDA) for decreasing the rate of drop in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), a chronic condition with few treatment options. The FDA has approved Actemra/RoActemra as the first biologic medication for the treatment of the condition. In April 2020, Boehringer Ingelheim received approval from the European Commission for nintedanib for the treatment of people with systemic sclerosis-associated interstitial lung disease (SSc-ILD). Regional Dominance: North America is estimated to rule the market for scleroderma diagnostics and therapeutics. Among the regions, North America is expected to dominate the global scleroderma diagnostics and therapeutics market. Major factors such as favorable reimbursement policies, heavy investment in R&D and drug development, and well-established healthcare infrastructure are boosting the growth of the market in this region. Additionally, the growing prevalence of the disease and technological advancement in the field of medical devices are also propelling the growth of the market in this region. Europe is anticipated to hold the second-most position in the market owing to an increase in initiatives and proactive awareness regarding the diagnostics for scleroderma. On the other hand, the market in Asia Pacific is likely to grow at the highest CAGR during the forecast period. This is majorly due to the wide and easy availability of generic & biosimilar immunosuppressants for the treatment of scleroderma. Global Scleroderma Diagnostics and Therapeutics Market is segmented as follows: Scleroderma Diagnostics and Therapeutics Market : By Diagnostic Test Type Outlook (2022-2028) Blood Tests Imaging Techniques Skin Biopsy Pulmonary Function Tests Electrocardiogram & Echocardiogram Scleroderma Diagnostics and Therapeutics Market : By Indication Outlook (2022-2028) Systemic Scleroderma Localized Scleroderma Scleroderma Diagnostics and Therapeutics Market : By Drug Class Outlook (2022-2028) Prostacyclin Analogues Endothelin Receptor Antagonists Phosphodiesterase 5 Inhibitors – PHA Immunosuppressors Analgesics Calcium Channel Blockers Others Scleroderma Diagnostics and Therapeutics Market : By Region Outlook (2022-2028) North America The U.S. Canada Europe France The UK Spain Germany Italy Rest of Europe Asia Pacific China Japan India South Korea Southeast Asia Rest of Asia Pacific Latin America Brazil Mexico Rest of Latin America Middle East & Africa GCC South Africa Rest of Middle East & Africa Press Release For Scleroderma Diagnostics and Therapeutics Market : Browse Other Related Research Reports from Zion Market Research Orthopedic Soft Tissue Repair Market - Global Industry Analysis : The global Orthopedic Soft Tissue Repair Market accrued earnings worth approximately 5.7 (USD Billion) in 2020 and is predicted to gain revenue of about 11.2 (USD Billion) by 2028, is set to record a CAGR of nearly 6.4% over the period from 2021 to 2028. Surgical Drainage Devices Market - Global Industry Analysis : The global Surgical Drainage Devices Market accrued earnings worth approximately 1.12 (USD Billion) in 2020 and is predicted to gain revenue of about 3.20 (USD Billion) by 2028, is set to record a CAGR of nearly 5.7% over the period from 2021 to 2028. Charcot-Marie-Tooth Disease Type I Market - Global Industry Analysis : The global charcot-marie-tooth disease type I market accounted for USD 480.72 in 2020 and is expected to reach USD 632.16 Million by 2028, growing at a CAGR of 3.5% from 2021 to 2028. Browse through Zion Market Research's coverage of the Global Healthcare Industry Follow Us on: LinkedIn | Twitter | Facebook About Us Zion Market Research is an obligated company. We create futuristic, cutting edge, informative reports ranging from industry reports, company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the client's needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to us—after all—if you do well, a little of the light shines on us . Contact Us: Zion Market Research 244 Fifth Avenue, Suite N202 New York, 10001, United States Tel: +49-322 210 92714 USA/Canada Toll Free No.1-855-465-4651 Email: [email protected] Website: Blog : | Logo: SOURCE Zion Market Research

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here. The biotech IPO market continues to charge full-steam ahead, with three more companies submitting their Nasdaq pitches to the SEC late Friday. The leaders among the pack are Adagio Therapeutics and Castle Creek Biosciences, each penciling in $100 million initially. Following up the rear is Eliem Therapeutics, jotting down $80 million for its raise estimate less than six months after launching out of RA Capital’s incubator. Friday’s filings come a week after Endpoints News’ conversation with Nasdaq official Jordan Saxe, who said the institution is in conversations with about 250 biotechs over the next 12 to 18 months. Per the Endpoints tally, the biotechs that have already priced have raised more than $10 billion combined. In addition to the three filings, French biotech Dynacure withdrew its application for an IPO after not pricing last week. Our own John Carroll took a deep look into Adagio’s high-profile quest for Nasdaq, which premium subscribers can read here. For the best of the rest, check out the news below. Castle Creek takes former Fibrocell programs public Ahead of a high-stakes Phase III readout in the second half of next year, Castle Creek is seeking to join the Nasdaq ranks. The Exton, PA-based biotech is focusing its efforts around a program called dabocemagene autoficel, or D-Fi, to treat recessive dystrophic epidermolysis bullosa, a sometimes fatal rare disease caused by a lack of collagen in the skin resulting in uncontrollable blistering. It’s a program that comes from the small acquisition of the biotech Fibrocell back in September 2019. Castle Creek has been working on rare diseases involving the skin since inception, with a former lead program in a broader epidermolysis bullosa population. But that candidate, a topical reformulation of the IL-1 beta anti-inflammatory drug diacerein, failed a Phase II study in October 2018. Jeff Aronin Co-founder Michael Derby had told Endpoints at the time that Castle Creek planned to advance the program into Phase III despite the failure, but it’s unclear if those plans ever came to fruition. Diacerein is no longer listed among the biotech’s pipeline, and its only mentions in the S-1 revolve around the old licensing agreements Castle Creek signed to re-engineer it as a topical drug. The biotech will continue to charge forward with the newer candidate, and plans to launch a second Phase III study before the end of 2021 in the dominant version of dystrophic epidermolysis bullosa. Though Castle Creek did not divulge exact figures in its SEC paperwork, it’s expected a significant portion of these funds will go toward the two studies and “pre-commercial” activities for D-Fi. There’s also another Fibrocell gene therapy for moderate to severe localized scleroderma in the works, and some IPO funds will be used to wrap up an ongoing Phase I/II trial, Castle Creek noted. The biotech comes from maven Jeff Aronin and his portfolio of companies under the Paragon umbrella. Once it prices, Castle Creek expects to trade under the ticker $CCBS. Eliem for a dream: From stealth to Nasdaq in four months Eliem Therapeutics is aiming to complete a quick rise to Nasdaq after launching at the end of March. There had been signs of an IPO after the biotech’s $60 million Series B, when company spokespeople declined interview requests ahead of the filing. Eliem likely represents one of the fastest Nasdaq pushes for biotech in recent memory, going from launch to filing for an IPO in less than four months. Should it price, Eliem will have a pipeline of four neuro assets led by a prodrug of an endocannabinoid known as palmitoylethanolamide, or PEA. The candidate is known as ETX-810, and CSO Valerie Morisset previously told Endpoints the biotech is looking at a variety of chronic pain indications. ETX-810 is currently in two Phase IIa studies looking at diabetic peripheral neuropathic pain and lumbosacral radicular pain. Data readouts are expected for the first half of 2022. Eliem is also developing a GABA-positive allosteric modulator, which the company is calling ETX-155. Researchers plan to take this program into two Phase IIa studies for major depressive disorder and hormone-related depressive disorders, as well as a Phase Ib trial for epilepsy. Data here are expected in the second half of 2022 and the first half of 2023, respectively. Both programs come from RA Capital, where the biotech was incubated. Both will also be the primary focuses for the IPO raise. Once Eliem goes public, it will trade under the ticker $ELYM.