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Teen Cured Of Rare Genetic Disorder: World's First Case

Highlights:
  • A pioneering medical team achieved a landmark by curing Kai, 13, of WILD syndrome and severe chylous ascites, rare genetic disorders
  • Dr. Mona Mossad's innovative approach and collaborative effort led to a groundbreaking treatment plan, a first in pediatric care
  • Kai's remarkable response to treatment underscores the dedication and expertise of the medical team at Staffordshire Children's Hospital
  • A team of innovative British doctors achieved a medical milestone by curing a teenager diagnosed with an extremely rare genetic disorder, WILD syndrome, and severe chylous ascites. Kai Xue, 13, was among just 21 individuals worldwide afflicted with WILD syndrome, a condition characterized by cell impairment, warts, and susceptibility to infections (1✔ ✔Trusted SourceTeen with Incredibly Rare Genetic Condition is Cured in World First By British DoctorsGo to source). Additionally, Kai battled severe chylous ascites, a life-threatening ailment causing 28 liters of lymphatic fluid accumulation in her abdomen. A Decade-Long Journey to Diagnosis and Treatment Ning Chen, Kai's mother, recounted a challenging journey of seeking treatment for her daughter, which included visits to various hospitals and even a trip to China. However, after more than a decade of uncertainty, Kai's condition was finally diagnosed by Dr. Mona Mossad, a renowned expert in lymphatic intervention. Dr. Mossad collaborated with skilled surgeons to develop a groundbreaking treatment plan, marking the first time such a procedure was offered to a child. 'Did you know?Only 21 people worldwide were diagnosed with WILD syndrome, making Kai's recovery a truly exceptional feat in medical history.#medicalinnovation #raredisorders #medindia' The medical team's comprehensive approach involved improving Kai's lymphatic drainage and performing intricate surgery to block lymphatic vessels in her liver. Despite the complexity of the case, Kai responded remarkably well to the treatment, eventually allowing her to return home to her family.Celebrating Kai's Recovery and the Medical Team's Dedication Dr. Yvonne Slater, Consultant Paediatric Gastroenterologist at Staffordshire Children's Hospital at Royal Stoke, expressed delight at Kai's positive response to treatment, emphasizing the collaborative effort of multiple medical teams involved in her care.

    Ning expressed heartfelt gratitude to the doctors and surgeons who played a pivotal role in her daughter's recovery, praising their dedication and expertise. She credited the entire medical team for their exceptional care and support throughout Kai's journey to recovery, underscoring their unwavering commitment to providing the best possible outcome for their patients.

    Reference:
  • Teen with Incredibly Rare Genetic Condition is Cured in World First By British Doctors - (https://www.Goodnewsnetwork.Org/teen-with-incredibly-rare-genetic-condition-is-cured-in-world-first-by-british-doctors/ )
  • Source-Medindia

    Galactosemia Treatment Market Expected To Double By 2032, Projected Growth At 6.8% CAGRMarketresearch.biz

    Marketresearch.Biz reports that the Galactosemia Treatment Market size is expected to be worth around USD 893.5 Mn by 2032 from USD 470.5 Mn in 2022, growing at a CAGR of 6.8% during the forecast period from 2023 to 2032.

    Overview of the Galactosemia Treatment Market

    Galactosemia, a rare genetic disorder, necessitates specialized treatments, making its market unique. Therapies aim to manage symptoms and prevent complications, driving innovation and investment in this niche market.

    Get Full PDF Sample Copy of Report (Including Full TOC, List of Tables & Figures, Chart) Click Here to Download a Sample Report: https://marketresearch.Biz/report/galactosemia-treatment-market/request-sample/

    Driving Factors of the Galactosemia Treatment Market
  • Advancements in Gene Therapy: Research into gene therapy offers promising avenues for addressing the underlying genetic mutations causing galactosemia.
  • Increasing Awareness: Growing awareness among healthcare professionals and patients about early diagnosis and treatment options fuels market growth.
  • Government Initiatives: Supportive policies and funding for rare disease research encourage pharmaceutical companies to invest in galactosemia treatments.
  • Technological Innovations: Advances in diagnostics and treatment modalities enhance the efficacy and accessibility of therapies for galactosemia patients.
  • Collaborative Research Efforts: Collaborations between academia, industry, and advocacy groups accelerate the development of novel treatments and therapeutic approaches.
  • Expanding Patient Pool: Improvements in screening programs and diagnostic techniques lead to a higher number of diagnosed cases, driving demand for treatment options.
  • Restraining Factors of the Galactosemia Treatment Market
  • Limited Patient Pool: The rarity of galactosemia restricts the market size, potentially reducing incentives for pharmaceutical companies to invest in research and development.
  • High Treatment Costs: The specialized nature of treatments for galactosemia may result in high therapy costs, posing financial challenges for patients and healthcare systems.
  • Regulatory Hurdles: Stringent regulatory requirements for drug approval in rare diseases can prolong the development timeline and increase investment risks for companies.
  • You can check In-Detail TOC from here: https://marketresearch.Biz/report/galactosemia-treatment-market/

    The Galactosemia Treatment Market report provides a comprehensive exploration of the sector, categorizing the market by type, application, and geographic distribution. This analysis includes data on market size, market share, growth trends, the current competitive landscape, and the key factors influencing growth and challenges. The research also highlights prevalent industry trends, market fluctuations, and the overall competitive environment.

    This document offers a comprehensive view of the Global Galactosemia Treatment Market, equipping stakeholders with the necessary tools to identify areas for industry expansion. The report meticulously evaluates market segments, the competitive scenario, market breadth, growth patterns, and key drivers and constraints. It further segments the market by geographic distribution, shedding light on market leadership, growth trends, and industry shifts. Important market trends and transformations are also highlighted, providing a deeper understanding of the market's complexities. This guide empowers stakeholders to leverage market opportunities and make informed decisions. Additionally, it provides clarity on the critical factors shaping the market's trajectory and its competitive landscape.

    Following Key Segments Are Covered in Our Report By Type
  • Classic Galactosemia
  • Clinical Variant Galactosemia
  • Biochemical Variant Galactosemia
  • By Route of Administration By Diagnosis
  • Genetic Testing
  • Blood tests
  • Urine tests
  • Others
  • By End-User
  • Hospitals and Clinics
  • Specialty Centers
  • Homecare Settings
  • By Distribution Channel
  • Hospital Pharmacy
  • Retail Pharmacy
  • Online Pharmacy
  • Others
  • Top Key Players in Galactosemia Treatment Market
  • F. Hoffmann-La Roche Ltd.
  • Mylan N.V.
  • Teva Pharmaceutical Industries Ltd.
  • Sanofi
  • Pfizer Inc.
  • GSK plc
  • Novartis AG
  • Merck & Co. Inc.
  • Abbvie, Inc.
  • AstraZeneca
  • Johnson & Johnson Private Limited
  • Bristol-Myers Squibb Company
  • Bayer AG
  • Boehringer Ingelheim International GmbH.
  • Other Key Players
  • Get Full PDF Sample Copy of Report (Including Full TOC, List of Tables & Figures, Chart) Click Here to Download a Sample Report: https://marketresearch.Biz/report/galactosemia-treatment-market/request-sample/

    Regional Analysis of Galactosemia Treatment Market
  • North America: With advanced healthcare infrastructure and high awareness levels, North America dominates the market. Stringent regulations and robust research initiatives drive innovation and market growth in this region.
  • Europe: Europe follows closely, benefitting from supportive healthcare policies and significant investments in rare disease research. Increasing prevalence and improving diagnostics contribute to market expansion.
  • Asia Pacific: The Asia Pacific region shows promising growth potential, driven by rising healthcare expenditure and improving healthcare infrastructure. Increasing awareness and expanding diagnostic capabilities drive market growth in countries like Japan, China, and India.
  • Middle East: While relatively smaller in size, the Middle East market for galactosemia treatment is expanding, supported by improving healthcare infrastructure and rising awareness about rare diseases among healthcare professionals and patients.
  • Africa: Africa faces challenges in healthcare access, but efforts to improve healthcare systems and raise awareness about rare diseases are gradually expanding the market. Partnerships with international organizations facilitate access to treatments in certain regions.
  • This regional analysis underscores the diverse dynamics influencing the Galactosemia Treatment Market across different geographical regions.

    For More Information or Qurey, Visit @ https://marketresearch.Biz/report/galactosemia-treatment-market/

    Growth Opportunities for Galactosemia Treatment Market
  • Increasing Diagnosis Rates: Rising awareness and improved diagnostic techniques lead to more cases being diagnosed, expanding the potential market for galactosemia treatments.
  • Technological Advancements: Ongoing advancements in gene therapy and other treatment modalities offer new avenues for effective management of galactosemia, driving market growth.
  • Emerging Markets: Untapped markets in developing regions present significant growth opportunities as healthcare infrastructure improves and awareness about rare diseases increases.
  • Collaborative Research: Collaborations between academia, pharmaceutical companies, and advocacy groups accelerate the development of innovative treatments, opening new avenues for market expansion.
  • Regulatory Support: Supportive regulatory frameworks and incentives for orphan drug development encourage investment in research and development for galactosemia treatments, fostering market growth.
  • Trending Factors in Galactosemia Treatment Market
  • Gene Therapy Advancements: Breakthroughs in gene therapy, including CRISPR-based technologies, hold promise for addressing the genetic mutations underlying galactosemia, driving market interest and investment.
  • Personalized Medicine: Increasing focus on personalized medicine allows for tailored treatment approaches based on individual patient profiles, enhancing treatment efficacy and patient outcomes in galactosemia management.
  • Patient Advocacy: Growing patient advocacy efforts raise awareness about galactosemia and advocate for improved access to treatments, influencing market dynamics and shaping healthcare policies.
  • Digital Health Solutions: Integration of digital health solutions, such as telemedicine and digital therapeutics, streamlines patient management and improves access to specialized care for galactosemia patients, driving market growth.
  • Investment in Rare Diseases: Increasing investment and funding for rare disease research and development create a conducive environment for innovation and market expansion in the galactosemia treatment sector.
  • These growth opportunities and trending factors highlight the dynamic landscape of the Galactosemia Treatment Market, reflecting ongoing advancements and evolving strategies to address the needs of patients with this rare genetic disorder.

    Our comprehensive Market research report endeavors to address a wide array of questions and concerns that stakeholders, investors, and industry participants might have. The following are the pivotal questions our report aims to answer:

    Industry Overview:

  • What are the prevailing global trends in the Galactosemia Treatment Market?
  • How is the Galactosemia Treatment Market projected to evolve in the coming years? Will we see a surge or a decline in demand?
  • Product Analysis:

  • What is the anticipated demand distribution across various product categories within Galactosemia Treatment?
  • Which emerging products or services are expected to gain traction in the near future?
  • Financial Metrics:

  • What are the projections for the global Galactosemia Treatment industry in terms of capacity, production, and production value?
  • Can we anticipate the estimated costs, profits, Market share, supply and consumption dynamics?
  • How do import and export figures factor into the larger Galactosemia Treatment Market landscape?
  • Strategic Developments:

  • What strategic initiatives and movements are predicted to shape the industry in the medium to long run?
  • Pricing and Manufacturing:

  • Which factors majorly influence the end-price of Galactosemia Treatment products or services?
  • What are the primary raw materials and processes involved in manufacturing within the Galactosemia Treatment sector?
  • Market Opportunities:

  • What is the potential growth opportunity for the Galactosemia Treatment Market in the forthcoming years?
  • How might external factors, like the increasing use of Galactosemia Treatment in specific sectors, impact the Market's overall growth trajectory?
  • Historical Analysis:

    What was the estimated value of the Galactosemia Treatment Market in previous years, such as 2022?

    Key Players Analysis:

  • Who are the leading companies and innovators within the Galactosemia Treatment Market?
  • Which companies are positioned at the forefront and why?
  • Innovative Trends:

  • Are there any fresh industry trends that businesses can leverage for additional revenue generation?
  • Market Entry and Strategy:

  • What are the recommended Market entry strategies for new entrants?
  • How should businesses navigate economic challenges and uncertainties in the Galactosemia Treatment Market?
  • What are the most effective Marketing channels to engage and penetrate the target audience?
  • Geographical Analysis:

  • How are different regions performing in the Galactosemia Treatment Market?
  • Which regions hold the most potential for future growth and why?
  • Consumer Behavior:

  • What are the current purchasing habits of consumers within the Galactosemia Treatment Market?
  • How might shifts in consumer behavior or preferences impact the industry?
  • Regulatory and Compliance Insights:

  • What are the existing and upcoming regulatory challenges in the Galactosemia Treatment industry?
  • How can businesses ensure consistent compliance?
  • Risk Analysis:

  • What potential risks and uncertainties should stakeholders be aware of in the Galactosemia Treatment Market?
  • External Impact Analysis:

  • How are external events, such as geopolitical tensions or global health crises (e.G., Russia-Ukraine War, COVID-19), influencing the Galactosemia Treatment industry's dynamics?
  • This report is meticulously curated to provide a holistic understanding of the Galactosemia Treatment Market, ensuring that readers are well-equipped to make informed decisions.
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    MarketResearch .Biz, a division of Prudour Pvt Ltd, excels in providing thorough Market research and analytical services. With a strong history of reliability, our company has established itself as a trusted consulting agency and a source for custom Market research insights. At MarketResearch .Biz, we recognize the diverse needs of our clients and are equipped to offer reports tailored to their specific requirements. Our dedication extends beyond standard practices, ensuring that we consistently deliver top-notch insights and a comprehensive view of the Market landscape to our clients.

    Mr. Lawrence JohnMarketresearch.Biz ( View More Related Report:

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    Does Ghee Hurt Heart Health?

    A dietitian explains its impact on this and more.

    Medically reviewed by Elizabeth Barnes, RDN

    Ghee is a form of butter commonly used in cooking for flavor and as a source of fat.

    Ghee has been around for centuries, with its original use rooted in the traditional medicine system of India, known as Ayurveda. However, limited clinical studies exist on ghee's purported benefits for heart and gut health and weight management.

    The following article covers the uses of ghee, its nutritional value, and safety considerations.

    knape / Getty Images

    Ghee clarified butter in jar What Is Ghee?

    Ghee is clarified butter but cooked longer to reduce milk solids further and ensure a nutty taste. Clarification occurs when milk solids and water separate from the fat.

    Ghee is produced by cooking down milk, cream, or butter to remove the water and milk solids. Heat causes the milk solids to caramelize and separate from the fat, imparting a nutty flavor to the liquid as the solids brown.

    Heat breaks down milk proteins like casein and lactose, giving ghee its unique nutrition profile. Unlike butter, individuals with lactose intolerance can consume ghee without issue. 

    In India, ghee is considered sacred and used in religious rituals. It is consumed throughout the country and is a key ingredient in cooking. Ghee is viewed as pure—anything cooked with ghee contains natural purity.

    Ayurveda emphasizes the therapeutic benefits of ghee, both alone and in combination with other medicinal components. It endorses and finds no harm in consuming ghee regularly, even daily.

    Ghee Nutrition

    Ghee has a nutrition profile similar to that of butter. One tablespoon (14 grams) of ghee provides the following nutrition:

  • Calories: 123 kilocalories (kcal)

  • Fat: 14 grams (g)

  • Vitamin A: 118 micrograms (mcg) retinol activity equivalents (RAE) and 17% of the daily value (DV)

  • Vitamin E: 0.4 milligrams (mg) and 3% of DV

  • Ghee is almost all fat (95.5%) and is less than 0.5% moisture.

    Nearly nine of the 14 grams of fat in a serving of ghee are from saturated fat. The Dietary Guidelines for Americans recommends that saturated fat should encompass no more than 10% of total intake. One tablespoon of ghee contributes more than one-third of the recommended saturated fat.

    However, ghee is also a significant source of conjugated linoleic acid (CLA), a polyunsaturated omega-6  fatty acid. CLA exists naturally in dairy products.

    Additional health benefits of CLA include the following effects:

    CLA supplements abound because of their potential to prevent chronic conditions such as metabolic syndrome (a group of conditions that together can contribute to heart disease, stroke, and type 2 diabetes).

    Fat-Soluble Vitamins in Ghee

    Ghee is a potent source of vitamin A, a fat-soluble nutrient, meaning it's absorbed with fats in your diet. Vitamin A is essential for the following processes: 

    Ghee is derived from milk, cream, or butter and contains other fat-soluble vitamins. The content of vitamins like D, E, and K varies depending on the brand of ghee.

    According to one study,  ghee's average vitamin D content is 11.42 IU per gram (2% of DV). Vitamin D serves many functions in the body. It promotes bone health, reduces inflammation, and supports immune function.

    Vitamin E is an essential antioxidant that protects cells from damage by unstable molecules called free radicals. Additionally, vitamin E supports immune health.

    Consuming fat-soluble vitamins within a fat source like ghee aids with nutrient absorption.

    Safety Profile

    Ghee's nutrition profile affects its safety concerns and health impact.

    Impact on Heart Health

    Limited research exists on the impact of ghee on heart health.

    Preliminary research in rats suggests that ghee reduces the risk of cardiovascular disease by lowering cholesterol and triglycerides and raising levels of high-density lipoprotein (HDL) cholesterol ("good cholesterol").

    Ghee is a source of conjugated linoleic acid (CLA). Researchers attribute ghee's benefit on rats' cholesterol levels to the antioxidant effects of CLA. CLA boosts the activity of enzymes that squelch free radicals. Moreover, CLA prevents fatty acid cell damage, which contributes to the development of cardiovascular disease.

    One study of people living in Pakistan observed no association between ghee and coronary artery disease (CAD) after adjusting for age, hypertension (high blood pressure), diabetes, body mass index (BMI), and smoking. Ghee neither increased nor decreased the risk of CAD.

    Still, the American Heart Association (AHA) recommends aiming for five to six percent of calories from saturated fat. The AHA cites significant evidence linking saturated fat with low-density lipoprotein (LDL) cholesterol ("bad cholesterol") and an increased risk of heart disease.

    However, additional research on the heart health benefits of ghee in humans is necessary.

    Impact on Gut Health

    Ghee is clarified, so it does not contain casein or lactose. Therefore, people who are lactose intolerant can consume ghee without issues.

    Moreover, some research states that ghee is safe for people with galactosemia. Galactosemia is a genetic condition in which the body cannot break down galactose, a sugar in dairy.

    Although Ayurveda promotes the benefit of ghee for the gut, minimal research supports ghee for gastrointestinal health.

    Impact on Weight Management

    Ghee contains medium-chain triglycerides (MCT), a type of fat that is more rapidly broken down and absorbed by the body. Research implies that MCTs are associated with weight loss. According to some studies, MCTs increase energy expenditure and contribute to feelings of fullness (satiety).

    One meta-analysis found that consuming MCTs instead of long-chain triglycerides (LCT) resulted in an average weight loss of 0.51 kilograms (kg), equal to about 1 pound, over 10 weeks. Opting for MCTs instead of LCTs can decrease waist and hip circumferences and body fat.

    Evidence yields mixed results on the effects of MCTs on appetite. Some studies show MCTs cause greater feelings of fullness and reduced food consumption, contributing to weight loss. Conversely, other research reveals no difference between MCTs and other fats for satiety (satisfied feeling of fullness). In some cases, MCTs even contribute to increased hunger.

    Ghee also contains conjugated linoleic acid (CLA). CLA supplements support fat and weight loss. However, it is unclear whether CLA in ghee produces similar results.

    Despite the potential effect of MCTs on weight, it is essential to moderate ghee consumption. Ghee is a calorically dense food, meaning it contributes a significant amount of calories in a small quantity. Ghee use can add up quickly and contribute to undesirable changes in weight and body composition.

    The Research on Ghee

    Limited research exists on the health benefits of ghee itself.

    Ghee and Wound Healing

    Ayurvedic and Indian folk medicine associate the use of ghee with wound healing. Preliminary animal studies show faster wound healing with ghee applied to the injury. Ghee appears to favorably alter the healing process by increasing the activity of cells involved in wound repair.

    According to some studies, gauze soaked in ghee and honey improves infected ulcers and incisions from cesarean sections (C-sections). Additional in vitro (nonhuman and nonanimal) research supports ghee and honey for scarless wound healing.

    Traditional medicine attributes the benefits of the honey-ghee mixture to its antimicrobial, moisturizing, deodorizing, and rapid healing properties. Moreover, some research indicates no adverse effects with honey-ghee dressing.

    Again, more research is needed on the benefits of ghee for wound health. Always consult a healthcare provider before treating wounds with any medications.

    Ghee and Blood Sugar

    One in vivo (animal) study showed that adding ghee to red or white rice at different cooking points impacted blood sugar response.

    Adding ghee to the rice after cooking produced the smallest rise in glucose (blood sugar) levels. Likewise, red rice with ghee added after cooking had less of an impact on blood sugar than white rice.

    It is well-established that pairing a fat source with a carbohydrate slows the release of glucose in the bloodstream. However, it is unclear whether ghee benefits blood sugar more compared to other fat sources.

    How to Use Ghee

    Ghee has a higher smoke point (465 degrees F) than butter (302 degrees F). Safflower, soybean, canola, and avocado oils have high smoke points similar to ghee. Use ghee interchangeably with these oils depending on the desired flavor profile of a dish.

    For example, use ghee instead of a high-smoke-point oil if you want sautéed vegetables to taste richer and nuttier.

    Due to its high smoke point, ghee is commonly used for frying.

    However, fried food in general should be limited because of its negative effects on health. One large meta-analysis of more than 500,000 people found that people who ate the largest quantities of fried food had a 28% increased risk of cardiovascular disease than those who consumed the smallest quantities.

    Still, ghee can be part of a healthy diet. It is advantageous for cooking at high temperatures, but use it sparingly because it is an energy-dense, high-fat food. When using ghee, be mindful of portion size. As ghee is almost entirely fat, a small portion contributes significant calories. A typical serving is 1 tablespoon.

    How to Make Ghee at Home

    Ghee can easily be prepared at home. To do so:

  • Simmer butter until it liquefies, produces a foam on top, and the milk solids separate.

  • Let the mixture sit for a few minutes off the stove for a robust flavor.

  • Skim the foam from the top and separate the milk solids from the gold-colored liquid (ghee) using a strainer or cheesecloth.

  • Store the ghee in an airtight container. Because of its high fat content, ghee solidifies at room temperature. It is also shelf-stable, so it is easy to keep on hand.

    Is Ghee Good for Me?

    Avoid ghee if you're allergic to it or its components (parts). Seek immediate medical attention if you have a severe allergic reaction (itching, hives, shortness of breath).

    Ghee is generally safe and well-tolerated. It can be a great alternative to butter for individuals sensitive to lactose or casein.

    However, individuals following a low-fat diet should minimize ghee consumption because of its high fat content.

    Discuss the use of ghee with your healthcare provider if you are at a high risk of cardiovascular disease.

    Summary

    Ghee is a dense clarified butter cooked longer for a nutty flavor. Its health benefits are promoted in Ayurveda. Several cuisines commonly use ghee. Its high smoke point makes it favorable for frying, sautéing, or cooking over high heat.

    While it has a rich history, limited research supports the benefits of ghee for heart and gut health, weight management, wound healing, and blood sugar. People with cardiovascular disease should be mindful of using it due to its saturated fat content.

    Still, when used for cooking, ghee can be part of a healthy diet and provide food with additional flavor.

    Read the original article on Verywell Health.

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