Autosomal dominant disorders

TSC Alliance Applauds Release of Updated International Guidelines for Tuberous Sclerosis Complex Diagnostic and Management Recommendations

Retrieved on: 
Tuesday, August 3, 2021

SILVER SPRING, Md., Aug. 3, 2021 /PRNewswire/ --Today, the TSC Alliance announced the publication of two new papers in the prestigious Pediatric Neurology: " Updated International Tuberous Sclerosis Complex Diagnostic Criteria and Surveillance and Management Recommendations " and " Beyond the Guidelines: How We Can Improve Healthcare for People with Tuberous Sclerosis Complex Around the World ."

Key Points: 
  • SILVER SPRING, Md., Aug. 3, 2021 /PRNewswire/ --Today, the TSC Alliance announced the publication of two new papers in the prestigious Pediatric Neurology: " Updated International Tuberous Sclerosis Complex Diagnostic Criteria and Surveillance and Management Recommendations " and " Beyond the Guidelines: How We Can Improve Healthcare for People with Tuberous Sclerosis Complex Around the World ."
  • Tuberous sclerosis complex (TSC) causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin and lungs.
  • "The TSC Alliance is thrilled to announce updated 'gold standards' for TSC diagnosis, surveillance and management," said President & CEO Kari Luther Rosbeck.
  • Additionally, TSC International , a consortium of organizations that support individuals with TSC around the world, penned the companion piece, "Beyond the Guidelines: How We Can Improve Healthcare for People with Tuberous Sclerosis Complex from Around the World."

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Huntington Disease

Retrieved on: 
Thursday, July 29, 2021

This news comes only one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA).

Key Points: 
  • This news comes only one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA).
  • This new designation completes the quartet of orphan designations for VO659 in HD and SCA in both the US and EU.
  • Patients affected by Huntington's disease experience motor disturbances, personality changes and dementia, leading to increasing disability, loss of independence and reduced survival.
  • VICO's antisense oligonucleotide platform (AON) is focusing on different forms of spinocerebellar ataxia (SCA) and Huntington Disease (HD).

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Huntington Disease

Retrieved on: 
Thursday, July 29, 2021

This news comes only one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA).

Key Points: 
  • This news comes only one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA).
  • This new designation completes the quartet of orphan designations for VO659 in HD and SCA in both the US and EU.
  • Patients affected by Huntington's disease experience motor disturbances, personality changes and dementia, leading to increasing disability, loss of independence and reduced survival.
  • VICO's antisense oligonucleotide platform (AON) is focusing on different forms of spinocerebellar ataxia (SCA) and Huntington Disease (HD).

Global Renal Cell Carcinoma Epidemiology and Patient Flow Report 2020-2035: Targeted Population for Research and Development, Commercialization - ResearchAndMarkets.com

Retrieved on: 
Thursday, July 15, 2021

The "Global Renal Cell Carcinoma Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Renal Cell Carcinoma Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com's offering.
  • Global Renal Cell Carcinoma Epidemiology and Patient Flow Analysis - 2021, provides Renal Cell Carcinoma epidemiology, demographics, and patient flow.
  • The research provides population data to characterize Renal Cell Carcinoma patients, history of the disease at the population level (Renal Cell Carcinoma prevalence, Renal Cell Carcinoma incidence) and at the clinical level (from diagnosis to treated patients).
  • Renal Cell Carcinoma patient flow: Renal Cell Carcinoma prevalence, diagnosed, and drug-treated patients
    Demographics: Renal Cell Carcinoma patients by age group, gender
    The data from this research will help executives:
    Establish basis for Renal Cell Carcinoma market sizing, assessing market potential, and developing drug forecast models
    Identify Renal Cell Carcinoma patients segments through age groups, gender, and disease sub-types
    Evaluate Renal Cell Carcinoma market opportunities, identify target patient population
    View source version on businesswire.com: https://www.businesswire.com/news/home/20210715005764/en/

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

Retrieved on: 
Tuesday, June 29, 2021

In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.

Key Points: 
  • In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.
  • Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases."
  • "We're excited to obtain orphan-drug designation from also the FDA, following the EC grant earlier this year.
  • The designation allows VICO to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

Retrieved on: 
Tuesday, June 29, 2021

In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.

Key Points: 
  • In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.
  • Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases."
  • "We're excited to obtain orphan-drug designation from also the FDA, following the EC grant earlier this year.
  • The designation allows VICO to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.

NeuBase Therapeutics Announces Speakers for Virtual R&D Day Taking Place on June 8th

Retrieved on: 
Wednesday, June 2, 2021

NeuBase will present new data for the Companys pipeline including myotonic dystrophy type 1 (DM1), Huntingtons disease (HD), and its new oncology program.

Key Points: 
  • NeuBase will present new data for the Companys pipeline including myotonic dystrophy type 1 (DM1), Huntingtons disease (HD), and its new oncology program.
  • Using the Companys proprietary PATrOL platform, NeuBase is designing precision genetic medicines to activate, silence, or edit genes, as appropriate, depending on the underlying genetic mutation driving disease.
  • This unifying platform capability is uniquely positioned to create a scalable pipeline of new medicines to address previously untreatable diseases.
  • Dr. Friedlander is an elected member of the prestigious American Society for Clinical Investigation and the Association of American Physicians.

2021 Global Friedreich's Ataxia Epidemiology and Patient Flow Report - ResearchAndMarkets.com

Retrieved on: 
Wednesday, May 12, 2021

b'The "Global Friedreich\'s Ataxia Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nThis research report provides Friedreich\'s Ataxia epidemiology, demographics, and patient flow.

Key Points: 
  • b'The "Global Friedreich\'s Ataxia Epidemiology and Patient Flow - 2021" report has been added to ResearchAndMarkets.com\'s offering.\nThis research report provides Friedreich\'s Ataxia epidemiology, demographics, and patient flow.
  • The data is presented by Pharma G7 countries including the US, France, Spain, Italy, UK, Japan.\nThe research provides population data to characterize Friedreich\'s Ataxia patients, history of the disease at the population level (Friedreich\'s Ataxia prevalence, Friedreich\'s Ataxia incidence) and at the clinical level (from diagnosis to treated patients).
  • It also helps to identify patient sub-groups (age, gender, sub-groups) to understand targeted population for research and development, commercialization.\nDemographics: Friedreich\'s Ataxia patients by age group, gender\nThe data from this research will help executives:\nEstablish basis for Friedreich\'s Ataxia market sizing, assessing market potential, and developing drug forecast models\nIdentify Friedreich\'s Ataxia patients segments through age groups, gender, and disease sub-types\nEvaluate Friedreich\'s Ataxia market opportunities, identify target patient population\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210512005433/en/\n'

uniQure to Participate in Multiple Upcoming Industry Conferences in May

Retrieved on: 
Monday, May 3, 2021

ET.

Key Points: 
  • ET.
  • The live webcast of the fireside chat can be accessed through the link displayed in the Investors & Newsroom section of the uniQure website.\nuniQure is delivering on the promise of gene therapy \xe2\x80\x93 single treatments with potentially curative results.
  • We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.
  • www.uniQure.com\n"

uniQure Announces Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Wednesday, April 28, 2021

b'LEXINGTON, Mass.

Key Points: 
  • b'LEXINGTON, Mass.
  • and AMSTERDAM, The Netherlands, April 28, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that five data presentations, of which two are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14.\n"Our presentations at ASGCT showcase our gene therapy expertise in hemophilia and Huntington\xe2\x80\x99s disease, as well as advantages of the AAV5 vector in dosing through pre-existing neutralizing antibodies,\xe2\x80\x9d stated Ricardo Dolmetsch, Ph.D.president of research and development at uniQure.
  • We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington\'s disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.
  • www.uniQure.com\n'