Rickets

Avanzanite Bioscience Expands Partnership With Advicenne in Additional European Countries

Retrieved on: 
Wednesday, October 18, 2023

Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a specialty pharmaceutical company with a corporate mission of boosting orphan drug access and unlocking their full potential in European markets, today announced that it has expanded its commercial partnership with Paris-based specialty pharmaceutical company Advicenne for Sibnayal®.

Key Points: 
  • Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a specialty pharmaceutical company with a corporate mission of boosting orphan drug access and unlocking their full potential in European markets, today announced that it has expanded its commercial partnership with Paris-based specialty pharmaceutical company Advicenne for Sibnayal®.
  • Today’s announcement extends Avanzanite’s exclusive commercialization and distribution rights into three additional European countries, including Ireland, Cyprus and Malta.
  • The condition leads to stunted growth in children, brittle bones or rickets, kidney stones and life-threatening hypokalemia.
  • Announced in February 2023 , the original geographic scope of the partnership included Germany, Austria, Switzerland and Greece.

WebMD Announces 16th Annual 2023 Health Heroes

Retrieved on: 
Wednesday, October 11, 2023

NEWARK, N.J., Oct. 11, 2023 /PRNewswire/ -- WebMD announced today the winners of the 2023 WebMD 16th annual Health Heroes Awards, which includes 4 categories: Lifelong Advocate, Patient Pioneer, Selfless Survivor and Driving Force.

Key Points: 
  • NEWARK, N.J., Oct. 11, 2023 /PRNewswire/ -- WebMD announced today the winners of the 2023 WebMD 16th annual Health Heroes Awards, which includes 4 categories: Lifelong Advocate, Patient Pioneer, Selfless Survivor and Driving Force.
  • "This year's Health Heroes are not only doing amazing things but they also have data or other measurable results that demonstrate the impact of their work," said Dr. John Whyte, Chief Medical Officer of WebMD.
  • With their findings in hand, they began meeting with public health leadership at the Centers for Disease Control and Prevention (CDC), National Institutes of Health (NIH), and World Health Organization (WHO) to forge a path toward more research on Long COVID.
  • For additional information on the 2023 WebMD Health Heroes, click here: https://www.webmd.com/healthheroes/default.htm

Should I be getting my vitamin D levels checked?

Retrieved on: 
Monday, October 2, 2023

Australia has seen a surge in vitamin D testing of children, with similar trends reported for adults around the world. GPs are now being urged not to test for vitamin D unnecessarily. So when is low vitamin D a potential concern? And when might you need to get your levels tested?How much vitamin D do we need?Vitamin D also has many other roles, including helping our immune defences and contributing to DNA repair and cell differentiation.

Key Points: 


Australia has seen a surge in vitamin D testing of children, with similar trends reported for adults around the world. GPs are now being urged not to test for vitamin D unnecessarily. So when is low vitamin D a potential concern? And when might you need to get your levels tested?

How much vitamin D do we need?

    • Vitamin D also has many other roles, including helping our immune defences and contributing to DNA repair and cell differentiation.
    • We can thank the sun for most of our vitamin D. A chemical in our skin called 7-dehydrocholesterol is converted to vitamin D after contact with UVB radiation from the sun.
    • While we get some vitamin D also through our diet, this makes a relatively small contribution.
    • It’s difficult to get much more than one-third of our daily vitamin D requirement from diet without supplementation.

Why avoid vitamin D deficiency?

    • Prolonged, severe vitamin D deficiency will lead to softening of bone tissue and cause diseases such as rickets (children) and osteomalacia (adults).
    • However, avoiding low concentrations is likely to be good for many aspects of health, with consistent evidence suggesting benefits for infectious diseases and autoimmune conditions such as multiple sclerosis.

Who is at risk of deficiency?

    • The two main reasons for vitamin D deficiency typically relate to: 1. not getting (enough) vitamin D through sun exposure.
    • Deficiency risk can be high for anyone who is housebound, such as older or disabled people in residential care.
    • The risk of deficiency increases if we always cover our skin carefully by modest cultural dress, and also dark skin pigmentation is known to reduce vitamin D synthesis.

Am I getting enough sun exposure?

    • In Australia, it is possible to get enough vitamin D from the sun throughout the year.
    • Read more:
      Why you need more Vitamin D in the winter

      During winter, catching enough sun can be difficult, especially if you spend your days confined indoors.

    • This is because sunlight exposure can only help produce vitamin D if the UVB rays reach us at the correct angle.

Why does excess vitamin D testing matter?

    • Excessive testing is also a waste of health-care resources, with one single test costing about the same as a years’ worth of vitamin D supplementation.
    • Very often, we can make relatively small changes to our lifestyles to reduce the risks of vitamin D deficiency.

Inozyme Pharma Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE)

Retrieved on: 
Tuesday, September 26, 2023

BOSTON, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive interim safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE, pseudoxanthoma elasticum).

Key Points: 
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.
  • Exploratory biomarker data were collected throughout the study to provide evidence of the potential for disease modification with ongoing treatment with INZ-701.
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • PK properties were consistent with those observed in the Phase 1/2 clinical trial in adults with ENPP1 Deficiency.

Avanzanite Bioscience Further Strengthens Leadership Team With Country Manager of Germany

Retrieved on: 
Tuesday, September 19, 2023

Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a specialty commercial-stage pharmaceutical company with a corporate mission of boosting orphan drug access and unlocking their full potential in European markets, today announced the appointment of Marc-Andre Goldschmidt as Country Manager of Germany.

Key Points: 
  • Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a specialty commercial-stage pharmaceutical company with a corporate mission of boosting orphan drug access and unlocking their full potential in European markets, today announced the appointment of Marc-Andre Goldschmidt as Country Manager of Germany.
  • Prior to Avanzanite, Mr. Goldschmidt served most prominently as national sales manager of neurology for Alexion Pharmaceuticals, where he filled senior sales positions with growing responsibility for six years.
  • Immediately prior to Avanzanite, Mr. Goldschmidt served as business unit director at Stada, where he led the team that achieved 75% market share growth in the neurology therapy area.
  • Earlier in his career, Mr. Goldschmidt was a district sales manager at Amgen with consistent and successful results.

Does an apple a day really keep the doctor away? A nutritionist explains the science behind 'functional' foods

Retrieved on: 
Thursday, August 10, 2023

We’ve all heard that an apple a day keeps the doctor away, but how true is that?

Key Points: 
  • We’ve all heard that an apple a day keeps the doctor away, but how true is that?
  • Apples are not high in vitamin A, nor are they beneficial for vision like carrots.
  • However, apples contain various bioactive substances – natural chemicals that occur in small amounts in foods and that have biological effects in the body.

Functional foods defined

    • Functional foods are not the same as superfoods.
    • The problem is that most of those claims are not based on scientific research, like the criteria for functional foods are.
    • In addition to the nutrients that our bodies need for growth and development, functional foods contain a variety of bioactive substances, each with a unique function in the body.
    • Research suggests that the carotenoids from foods and the other categories of bioactive substances may help prevent certain cancers and improve heart health.

History of the functional food movement

    • From the early 1900s to the 1970s, nutrition research focused on vitamin deficiencies.
    • In 1980, the U.S. government published the first dietary guidelines that encouraged people to avoid fat, sugar and salt.
    • Public health messaging encouraged people to replace fatty foods with starchy foods such as breads and pasta.

Japan’s focus on foods for health

    • To correct this problem, Japan became the first country to introduce the concept of functional foods in the 1980s.
    • Today, Japan uses the phrase “Food for Specialized Health Uses” for products that can be scientifically shown to promote health.
    • Japan has more than 1,000 foods and beverages approved as food for specialized health uses, such as hypoallergenic rice.

The bioactive components in apples

    • An apple’s natural dietary fibers are one of the bioactive components that lead to its being classified as a functional food.
    • Pectin functions to reduce the amount of sugar and fat that is absorbed into the body.
    • In addition, apples contain high amounts of natural chemicals known as polyphenols that have vital roles in promoting health and reducing chronic disease.
    • Because they are mainly in the peel, whole apples are better sources of polyphenols than juice or applesauce.

Revisiting the original question

    • One U.S. team analyzed the apple-eating patterns and number of doctor visits among more than 8,000 adults.
    • Once adjusted for demographic and health-related factors, the researchers found that the daily apple eaters used marginally fewer prescription medications than the non-apple eaters.
    • A group of European researchers found that eating two apples a day improved heart health in 40 adults.
    • And Brazilian investigators found that eating three apples daily improved weight loss and blood glucose levels in 40 overweight women.

Vitamin D Market is expected to generate a revenue of USD 2.07 Billion by 2030, Globally, at 6.45% CAGR: Verified Market Research®

Retrieved on: 
Wednesday, January 25, 2023

JERSEY CITY, N.J., Jan. 25, 2023 /PRNewswire/ -- Verified Market Research® recently published a report, "Vitamin D Market" By Analog Type (Vitamin D2, Vitamin D3), By Application (Animal feed & pet food, Functional food & beverages, Personal care), By End-User (Children, Adults, Pregnant Women), and By Geography.

Key Points: 
  • Eggs, sea food like fish, and dairy products all contain vitamin D. Vitamin D also makes the brain and neurological systems strong.
  • Ergocalciferol (vitamin D2) and cholecalciferol are the major two types of vitamin D. (vitamin D3).
  • The "Global Vitamin D Market" is mainly bifurcated into sub-segments which can provide classified data regarding the latest trends in the market.
  • The "Global Vitamin D Market" study report will provide valuable insight with an emphasis on the global market.

Vitamin D Global Market Report 2023: Increasing Demand for Vitamin D Formulation in Personal Care Boosts Sector - ResearchAndMarkets.com

Retrieved on: 
Thursday, January 12, 2023

The global vitamin D market is expected to grow at a CAGR of 8.49% during 2022-2027.

Key Points: 
  • The global vitamin D market is expected to grow at a CAGR of 8.49% during 2022-2027.
  • The vitamin D market is highly characterized by the increasing demand for supplements and functional foods.
  • Vendors are also tapping vitamin D3's potential to encourage beauty and health, which additionally contributes to the vitamin D market growth.
  • Such factors propel the awareness of Vitamin D, thus boosting the Vitamin D Market share globally.

Inozyme Pharma Announces Peer-Reviewed Publication Revealing Increased Prevalence of ENPP1 Deficiency

Retrieved on: 
Monday, December 5, 2022

BOSTON, Dec. 05, 2022 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced the publication of an article titled “Estimation of ENPP1 Deficiency Genetic Prevalence Using a Comprehensive Literature Review and Population Databases” in the Orphanet Journal of Rare Diseases. Leading disease experts Carlos Ferreira, M.D., of the National Institutes of Health (NIH) and Frank Rutsch, M.D., of Münster University Children’s Hospital, together with Genomenon, an AI-driven genomics company, analyzed the latest genomics data and found the estimated prevalence of ENPP1 Deficiency to be 1 in 64,000 pregnancies, more than tripling the prior estimate1.

Key Points: 
  • “Our medical field team’s disease awareness, education, and patient identification efforts suggested that the number of patients with ENPP1 Deficiency has been vastly underestimated.
  • “Given the higher prevalence of ENPP1 Deficiency, it becomes obvious that many affected individuals remain undiagnosed.
  • “The prevalence data were gathered using a comprehensive database of genomic evidence, providing an unprecedented view into how many people are born with ENPP1 Deficiency worldwide.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

New Study Shows Comprehensive Genomic Evidence is Critical for Calculating Rare Disease Prevalence

Retrieved on: 
Thursday, December 15, 2022

ANN ARBOR, Mich., Dec. 15, 2022 /PRNewswire-PRWeb/ -- Genomenon, Inc., an AI-driven genomics company, today announced the publication of a paper in the Orphanet Journal of Rare Diseases that calculates the prevalence of ENPP1 deficiency—a disease claiming more than half of all infants within the first few months of life—to be more than triple the prior estimate. The study, conducted by the Genomenon scientific team and leading disease experts from the National Institutes of Health (NIH) and Münster University Children's Hospital, highlights a core strength of Genomenon's rigorous methodology for identifying and classifying genetic data needed to improve rare disease prevalence calculations.

Key Points: 
  • The study, conducted by the Genomenon scientific team and leading disease experts from the National Institutes of Health (NIH) and Mnster University Children's Hospital, highlights a core strength of Genomenon's rigorous methodology for identifying and classifying genetic data needed to improve rare disease prevalence calculations.
  • "Discovery of this higher prevalence rate aptly demonstrates how our exhaustive knowledgebase of expertly curated genomic evidence supports pharmaceutical companies that are pioneering development of therapies for orphan diseases."
  • Traditional methods for estimating prevalence rely on clinical data, which can be unreliable for rare diseases such as ENPP1 deficiency that are difficult to diagnose.
  • Genomenon is an AI-driven genomics company focused on making genomic information actionable for patients with rare genetic diseases and cancer.