Tay–Sachs disease

World's first field-installed, clinically approved parallel transmit 7T MRI scanner resides at Auburn University

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Tuesday, March 26, 2024
College, Epilepsy, Physics, Friends, Brain, MRI, Cell, Terra, Multiple sclerosis, Magnetic resonance imaging, Homeostasis, Biomedical Research, Post-traumatic stress disorder, Intelligence, Patient, Physiology, Physician, Education, DSM-IV codes, Tay–Sachs disease, Research, Gold, History, Economic development, Biomarker, Auburn University, Light, Sodium, Computer engineering, Electricity, Parkinson's disease, Cognition, Veterinary medicine, Liberal arts education, Multimedia, Disorder, Emotion, Engineering, Food, Medical imaging

Additionally, with dedicated radiofrequency sodium imaging coils and parallel transmit technology, the scanner offers expanded imaging capabilities.

Key Points: 
  • Additionally, with dedicated radiofrequency sodium imaging coils and parallel transmit technology, the scanner offers expanded imaging capabilities.
  • These areas represent an intersection between the needs of the MRI research community, particularly in 7T imaging, and existing expertise and strengths of Auburn University.
  • A stark difference between the old and new 7T scanners involve parallel transmit technology and sodium imaging capability.
  • "If it wasn't for them, we wouldn't have the 7T, and we are indebted to them for their dedication and commitment to Auburn.

National Tay-Sachs & Allied Diseases Association Hosts First of Its Kind Drug Development Meeting for GM2

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Thursday, February 1, 2024
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BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.

Key Points: 
  • BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.
  • This input can inform FDA's decisions and oversight during drug development and their review of marketing applications.
  • Nearly 30 people will share their stories and experiences with infantile, juvenile, or late onset GM2, including adult patients, parents, caregivers, and bereaved family members.
  • This virtual meeting will be livestreamed via YouTube, open to the public, free to attend, but advance registration is required.

Therapeutic Solutions International Announces Paradigm Shifting Immunotherapy Product: Tolerogenic Neutrophils

Retrieved on: 
Friday, September 29, 2023
Stem Cells, Biotechnology, Practice Management, Health, General Health, Oncology, Other Science, Research, Infectious Diseases, Genetics, Science, Neutrophil, Tay–Sachs disease, Pathology, TSOI, Inflammation, Cell, Stem cell, Transplant, Publishing, Patent, Mouse, Bacteria, Virus, Vaccine

Importantly the injected cells allow other cells from the same donor to not be rejected, allowing for transplantation of therapeutic cells.

Key Points: 
  • Importantly the injected cells allow other cells from the same donor to not be rejected, allowing for transplantation of therapeutic cells.
  • As someone who has spent many years believing and publishing that the most potent tolerogenic cell is the immature dendritic cell.
  • “We are especially thankful to Tatiana Lebedeva who came up with the idea to investigate the tolerogenic potential of in vitro generated neutrophils and supported the experiments.
  • “Therapeutic Solutions International is focused on product development and concurrently elucidating biological mechanisms of our products, which allow for continuous improvement and credibility in the medical community,” said Timothy Dixon, President, and CEO.

Anticipated Surge in Global Gene Panel Market: Fuelled by Rising Prevalence of Genetic Disorders – ResearchAndMarkets.com

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Friday, July 28, 2023
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The global gene panel market is poised for impressive growth during the forecast period, driven by the increasing prevalence of genetic disorders such as Down syndrome, sickle cell disease, Tay-Sachs disease, and others.

Key Points: 
  • The global gene panel market is poised for impressive growth during the forecast period, driven by the increasing prevalence of genetic disorders such as Down syndrome, sickle cell disease, Tay-Sachs disease, and others.
  • The demand for gene panel testing surges in response to the increasing disorders caused by genetic mutations.
  • Genetic disorders are currently prevalent in the geriatric population, significantly supporting the expansion of the gene panel market.
  • The United States leads the global gene panel market due to the growing demand for gene panel techniques in response to the rising cancer cases worldwide.

Quest Launches Consumer-Initiated Genetic Test on questhealth.com to Deliver Personalized, Actionable Health Risk Insights

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Tuesday, July 11, 2023
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SECAUCUS, N.J., July 11, 2023 /PRNewswire/ -- Quest Diagnostics (NYSE: DGX), the leader in diagnostic information services, today announced the launch of its first consumer-initiated genetic test, now exclusively available through the company's consumer health business at questhealth.com. Called Genetic Insights, the new offering helps people understand their potential risk of developing certain inheritable health conditions, with advanced technology and end-to-end support that includes personalized health reports and access to genetic counseling.

Key Points: 
  • Called Genetic Insights , the new offering helps people understand their potential risk of developing certain inheritable health conditions, with advanced technology and end-to-end support that includes personalized health reports and access to genetic counseling.
  • "Genetic Insights goes beyond the typical consumer genetic test.
  • Based on the results of this test and personal or family health history, an individual's physician may suggest additional genetic testing.
  • The Genetics Insights test does not identify genetic risk markers of all known inheritable diseases.

New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease

Retrieved on: 
Wednesday, June 28, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Ignition, Partnership, GM2, Glycogen storage disease, Hope, Multimedia, AAV, Forge, Tay–Sachs disease, CDMO, CGMP, Patient, Entrepreneurship, HEK293, Pharmaceutical industry, Hearth

View the full release here: https://www.businesswire.com/news/home/20230628556153/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230628556153/en/
    “We are proud to be the CDMO partner for New Hope Research Foundation, an organization that is significantly advancing a gene therapy approach to provide hope for patients suffering from Tay-Sachs disease,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics.
  • “At Forge, we are ready to partner with companies, nonprofit organizations, and gene therapy developers around the world, working towards the mission we share to bring potentially life-saving gene therapies to patients.”
    Through this partnership, Forge will provide adeno-associated virus (AAV) process development, analytical services, and cGMP manufacturing to New Hope Research Foundation.
  • The Foundation will leverage Forge’s platform processes, including its proprietary HEK293 suspension Ignition Cells™, to accelerate the initial production.
  • All development and AAV manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy facility in Columbus, Ohio.

New AI Tool Makes Speedy Gene-Editing Possible

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Thursday, January 26, 2023
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Other conditions are caused, not by a mistake in the code itself, but by problems in how the cellular machinery reads DNA (epigenetics).

Key Points: 
  • Other conditions are caused, not by a mistake in the code itself, but by problems in how the cellular machinery reads DNA (epigenetics).
  • Similarly, zinc fingers can also hook onto transcription factors and pull them toward a gene segment in need of regulation.
  • The study authors' new technology, called ZFDesign, overcomes this obstacle by using artificial intelligence (AI) to model and design these interactions.
  • The model is based on data generated by the screen of nearly 50 billion possible zinc finger-DNA interactions in the researchers' labs.

Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, & Azafaros - ResearchAndMarkets.com

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Monday, October 24, 2022
Health, Pharmaceutical, News, GM2 gangliosidoses, Therapy, Glucose, GSL, Clinical trial, NDA, HIV disease progression rates, Sanofi, II, Technology, III, Discovery, Tay–Sachs disease, Cell death, Acquisition, Safety, Brain, Cerebellum, Pharmaceutical industry, Sandhoff disease

The "Sandhoff disease - Pipeline Insight, 2022," report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape.

Key Points: 
  • The "Sandhoff disease - Pipeline Insight, 2022," report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape.
  • The assessment part of the report embraces, in depth Sandhoff disease commercial assessment and clinical assessment of the pipeline products under development.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Sandhoff disease R&D.
  • The companies which have their Sandhoff disease drug candidates in the most advanced stage, i.e.

Insights on the Enzyme Replacement Therapy Global Market to 2028 - Players Include Sanofi, BioMarin Pharmaceutical and Takeda Pharmaceutical - ResearchAndMarkets.com

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Monday, September 12, 2022
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The "Enzyme Replacement Therapy Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Enzyme Type, Therapeutic Conditions, Route of Administration, and End User" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Enzyme Replacement Therapy Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Enzyme Type, Therapeutic Conditions, Route of Administration, and End User" report has been added to ResearchAndMarkets.com's offering.
  • The enzyme replacement therapy market is expected to grow from US$ 9,673.39 million in 2021 to US$ 15,184.70 million by 2028; it is estimated to grow at a CAGR of 6.8% from 2022 to 2028.
  • The growth of the global enzyme replacement therapy market is attributed to the rising prevalence of lysosomal storage diseases (LSDs) and rapid regulatory approval with other marketing benefits for drug with orphan drug designation.
  • Enzyme replacement therapy (ERT) is performed to treat congenital enzyme deficiencies by using enzymes or proteins having enzymatic activity.

Former Olympian Gail Devers, Emily Rapp Black, Jim Geraghty Among Speakers Announced for Global Genes 11th Annual RARE Patient Advocacy Summit

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Tuesday, August 9, 2022
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Global Genes , a leading rare disease patient advocacy organization, today announced the 2022 RARE Patient Advocacy Summit .

Key Points: 
  • Global Genes , a leading rare disease patient advocacy organization, today announced the 2022 RARE Patient Advocacy Summit .
  • In its 11th year, the Summit brings together rare disease patients, healthcare professionals, researchers, advocates and allies to connect, share experiences and resources, and gather insights about the latest rare disease innovations.
  • The Summit will be held September 12-14, 2022, both in-person in San Diego, CA, and virtually.
  • We are living in a time where connection is vitally important, said Craig Martin, CEO of Global Genes.