Development of the nervous system

Baylor Genetics Announces Neurodevelopmental Disorders Test Panel to Help Healthcare Providers Diagnose Patients with Intellectual Disabilities or Developmental Delays

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Tuesday, September 12, 2023
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The panel analyzes a clinically curated and targeted set of 236 genes to help healthcare providers and patients achieve a prompt, accurate genetic diagnosis and a better understanding of prognosis and anticipated disease progression.

Key Points: 
  • The panel analyzes a clinically curated and targeted set of 236 genes to help healthcare providers and patients achieve a prompt, accurate genetic diagnosis and a better understanding of prognosis and anticipated disease progression.
  • Baylor Genetics leveraged its genetic expertise and robust datasets to create the Neurodevelopmental Disorders Panel, which joins a growing catalog of disease-specific panels.
  • The panel is performed on an exome backbone capable of extracting a specific subset of genes for clinical reporting.
  • If panel results are negative, healthcare providers may request Whole Exome Sequencing allowing for broader analysis to help make a genetic diagnosis.

Zynerba Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results and Operational Highlights

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Tuesday, March 28, 2023
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DEVON, Pa., March 28, 2023 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, today reported financial results for the fourth quarter and full year ended December 31, 2022, and provided an overview of recent operational highlights and a pipeline update.

Key Points: 
  • Published data in the Journal of Neurodevelopmental Disorders describing the role of the endocannabinoid system and cannabidiol therapy in FXS.
  • Research and development expenses were $5.5 million for the fourth quarter of 2022, including stock-based compensation of $0.5 million.
  • General and administrative expenses were $3.2 million in the fourth quarter of 2022, including stock-based compensation expense of $0.5 million.
  • Net loss for the fourth quarter of 2022 was $8.0 million, with basic and diluted loss per share of $(0.18).

IAMA Therapeutics to Present Data on Its Epilepsy Program at American Academy of Neurology Annual Meeting (2023)

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Monday, March 13, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Other Health, MBA, Epilepsy, DSM-IV codes, AAN, IAMA, American Academy, Treatment, Therapy, Trial of the century, Safety, Development of the nervous system, Pharmaceutical industry

"IAMA-6 is one of several selective chloride modulators in IAMA’s portfolio that has been successfully formulated for potential clinical study as an oral formulation.

Key Points: 
  • "IAMA-6 is one of several selective chloride modulators in IAMA’s portfolio that has been successfully formulated for potential clinical study as an oral formulation.
  • Preclinical data demonstrate potential efficacy and safety of IAMA-6 at low, chronic dosing, in animal models of focal and pharmacoresistant epilepsies."
  • said Andrea P. Malizia, Ph.D., MBA, Chief Executive Officer at IAMA Therapeutics.
  • "In addition, development efforts will continue to characterize other candidates with a focus on reducing behavioral and cognitive comorbid conditions of pediatric epilepsy."

Therapeutic Targeting of the Microbiome for Neurodevelopmental Disorders - A Free Webinar from the Brain & Behavior Research Foundation

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Wednesday, February 8, 2023
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New York, Feb. 08, 2023 (GLOBE NEWSWIRE) -- The Brain & Behavior Research Foundation (BBRF) is hosting a free webinar, “Therapeutic Targeting of the Microbiome for Neurodevelopmental Disorders” on Tuesday, February 14, 2023, at 2:00 pm EST.

Key Points: 
  • New York, Feb. 08, 2023 (GLOBE NEWSWIRE) -- The Brain & Behavior Research Foundation (BBRF) is hosting a free webinar, “Therapeutic Targeting of the Microbiome for Neurodevelopmental Disorders” on Tuesday, February 14, 2023, at 2:00 pm EST.
  • The presenter will be Shelly A. Buffington, Ph.D., Assistant Professor, Department of Neuroscience, Cell Biology, & Anatomy at the University of Texas Medical Branch.
  • Her lab is also investigating the therapeutic potential of targeting the maternal gut microbiome to reduce risk for neurodevelopmental disorders in offspring.
  • The webinar is hosted by Jeffrey Borenstein, M.D., President & CEO of the Brain & Behavior Research Foundation, and host of the public television series Healthy Minds.

Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting

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Friday, December 2, 2022
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A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.

Key Points: 
  • A total of seven posters will be presented at the American Epilepsy Society (AES) 2022 Annual Meeting, December 2-6.
  • Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life.
  • STK-001 represents an entirely new approach to treatment, one that aims to treat the syndrome, not just the seizures.
  • Topline data from a combined interim analysis of the Phase 1/2a MONARCH and ADMIRAL studies showed single and multiple doses of STK-001 up to 45mg were well-tolerated.

Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Program at the American Epilepsy Society 2022 Annual Meeting

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Tuesday, November 29, 2022
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The company is advancing STK-001 as potentially the first medicine to treat the underlying cause of Dravet syndrome.

Key Points: 
  • The company is advancing STK-001 as potentially the first medicine to treat the underlying cause of Dravet syndrome.
  • Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP.
  • STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials.
  • Stokes first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy.

COMBINEDBrain Received the Global Genes 2022 Health Equity RARE Patient Impact Grant

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Thursday, September 29, 2022
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NASHVILLE, Tenn., Sept. 29, 2022 (GLOBE NEWSWIRE) -- COMBINEDBrain (Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders), a non-profit organization of patient advocacy groups representing rare genetic neurodevelopmental disorders, is pleased to announce they have been awarded a Global Genes Health Equity RARE Patient Impact Grant which will be used to translate the ClinGen Health Surveys into French, German, and Italian, in addition to Spanish and English which are currently available.

Key Points: 
  • NASHVILLE, Tenn., Sept. 29, 2022 (GLOBE NEWSWIRE) -- COMBINEDBrain (Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders), a non-profit organization of patient advocacy groups representing rare genetic neurodevelopmental disorders, is pleased to announce they have been awarded a Global Genes Health Equity RARE Patient Impact Grant which will be used to translate the ClinGen Health Surveys into French, German, and Italian, in addition to Spanish and English which are currently available.
  • For individuals and families facing a rare disease, the journey is already incredibly difficult, said Craig Martin, CEO of Global Genes.
  • The Global Genes health equity grant will help to break down this language barrier and improve health equity in research and treatment of rare neurodevelopmental disorders.
  • COMBINEDBrain non-profit 501c3 organization with a mission to speed the path to clinical treatments for people with severe rare genetic neurodevelopmental disorders: https://combinedbrain.org

Grant Paves Way for Imperial Valley High School Students to Enter Developmental Health Field

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Thursday, March 10, 2022
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SAN DIEGO, March 10, 2022 /PRNewswire/ -- Fred Finch Youth & Family Services (FF) announced today that it had received a grant from the Foundation for Developmental Disabilities (FDD) to spearhead a pilot program supporting high school students in Imperial Valley a highly underserved and underrepresented community.

Key Points: 
  • SAN DIEGO, March 10, 2022 /PRNewswire/ -- Fred Finch Youth & Family Services (FF) announced today that it had received a grant from the Foundation for Developmental Disabilities (FDD) to spearhead a pilot program supporting high school students in Imperial Valley a highly underserved and underrepresented community.
  • The program model called BEST (Behavior Education and Skills Training), will assist 10 Southwest High School seniors from El Centro, CA who are looking to learn more about careers in behavioral health this summer.
  • The BEST program will provide participants with training in understanding Autism, Neurodevelopmental disabilities, Applied Behavior Analysis, and basic medical and mental health terminology.
  • "The developmental disability fields are truly impacted right now and this program is a step in helping ease the pressures in Imperial Valley."

Stoke Therapeutics Announces Presentations from the Company’s Dravet Syndrome Program at the American Epilepsy Society 2021 Annual Meeting

Retrieved on: 
Friday, November 19, 2021
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At Stoke, our goal is to develop the first medicine to target the underlying cause of Dravet syndrome, a severe and progressive genetic epilepsy, said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics.

Key Points: 
  • At Stoke, our goal is to develop the first medicine to target the underlying cause of Dravet syndrome, a severe and progressive genetic epilepsy, said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics.
  • Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP.
  • STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials.
  • The Companys first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy.

ORYZON to Present New Clinical Data and Corporate Updates at International Conferences in September and October

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Wednesday, September 22, 2021
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Oryzon will present preliminary data from the ongoing collaboration with INGEMM in Phelan McDermid Syndrome (PMS) at the International Conference on Autism, ICA-2021, which will be held on September 23-24.

Key Points: 
  • Oryzon will present preliminary data from the ongoing collaboration with INGEMM in Phelan McDermid Syndrome (PMS) at the International Conference on Autism, ICA-2021, which will be held on September 23-24.
  • The communication entitled "Phenotype and Psychometric Characterization of Phelan McDermid Syndrome Patients" will be presented by Dr. Carlos Buesa, Oryzons CEO.
  • Executive directors of the company will attend the BIO Spain 2021 event, which will take place September 27-October 1.
  • The company will moderate a KOL panel discussion on precision medicine therapeutic approaches for CNS disorders and the potential of LSD1 inhibitors like vafidemstat.