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By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.
Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.
This will be launched today at a workshop taking place in Beijing, China. The toolkit will be launched during a five-day training workshop in Beijing, China, 23 – 27 November, organized by the Chinese Centre for Disease Control and Prevention and the Chinese Anti-TB Association, in collaboration with TDR and the WHO Global TB Programme.
Workshop addresses oncology dose optimization across full span of development In a series of broad-ranging, frank discussions, attendees at a joint FDA-American Association of Cancer Research (AACR) workshop worked through opportunities and challenges for dose optimization across the span of cancer drug development activities.
Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”
Offer not valid on workshop only or academic/non-profit registrations. Harness data-driven tech offerings to combat the urgent public health crisis and provide patients and stakeholders with unrivaled support services in an all-encompassing state-of-the-art hub. Drug Channels, or any of its employees.
He started to imagine how the deadly and contagious disease, if confirmed, might spread to half the city’s population. Eight of the 20 patients died, but the spread of the disease in Nigeria stopped there. Blood and urine samples from the man were waiting for Happi in his lab. Happi felt chilled. They called the idea Sentinel.
Knowing they could not hold PFDD meetings for every disease ( there are over 10,000 rare diseases alone ), they decided to turn PFDD meetings over to disease advocacy organizations, calling them Externally-Led Patient Focused Drug Development (EL-PFDD) meetings. There have been challenges in gene therapy development.
Access USA will unite PAP – Patient Assistance & Access Programs, Hub and Specialty Pharmacy Models East and Rare Disease Innovation & Partnership Summit, three industry-leading access conferences under one roof for one week of collaborative discussions and opportunities to expand your network and establish powerful partnerships.
Learn more Common Metabolic Diseases Knowledge Portal Aggregated genetic, epigenomic, and computational results for multiple metabolic diseases and traits. Learn more Single Cell Portal Single-cell -omic datasets from >400 research studies, representing several species, organ systems, and disease areas.
The Scientific Communication course, BLD sessions, Faculty Breakfast, and workshops helped us develop into better versions of ourselves and scientists. Project: Surveillance of H5N1 virus in milk in response to outbreaks in US dairy cattle Mentors: Elyse Stachler and Kyle Pacheco, Infectious Diseases and Microbiome Program
Attorney’s Office for the Eastern District of Pennsylvania Suzanne Marinakos, Director, Patient Services Operations- Rare Disease , Novo Nordisk Snigdha Santra, Director, Business Insights and Strategy , Chugai Pharmaceuticals Kinshuk Saxena, Pharm.D., Offer not valid on workshop only or academic/non-profit registrations.
Early in my education, I developed an interest in understanding how chemical entities derived from natural resources could be modified and used to fight specific diseases. We host Empowering Confidence Workshops where girls ages 13-18 get paired with women mentors, like me, and leaders in STEM.
This study represents the first ever to explore and describe the experiences of INFOSAN members with respect to their participation in network activities to improve global food safety and prevent foodborne diseases and to describe the characteristics of INFOSAN as a community of practice. Kuwait National Virtual INFOSAN Workshop.
For example, innate immune peptides being imitated by SARS-CoV-2 fragments have a special ability to organise double stranded RNA into structures that strongly amplify activation of the human innate immune system in autoimmune diseases. He currently serves on the Editorial Board of Physical Review E.
2 October, from 5:30pm The Learning Zone CV Workshops in person Get personalised advice on your CV from our industry experts. This workshop is designed to help you highlight your skills effectively. This fast-paced, dynamic event will leave you with new contacts and confidence in your networking abilities.
Our lead asset is in development for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare disease with no approved treatments, so we have prioritized incorporating patient and care partner voices across every stage of development. Building Community Understanding the biology of the disease is the beginning.
FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. a disease or condition is defined as rare by the Orphan Drug Act (ODA) if it affects fewer than 200,000 people.
Despite the growing adoption of high-content screening (HCS), analyzing the complex imaging data produced by these systems can take weeks and typically requires hands-on programming by data scientists.
He said that last year, more than 1,000 personalized cell and gene therapeutics were in clinical trials worldwide, and over 80% of these were for treating oncological diseases. The learnings from the workshop were that centers should form quality risk management working groups to explore the risk of these compounds.
The implication of this work is that we have an unprecedented opportunity to advance a single tRNA medicine to unify treatment across a wide range of diseases with the same underlying genetic mutation, enabling us to provide medicines to patients, especially those with rare or ultra-rare diseases, with no or limited treatment options.
By offering a range of services, such as vaccination clinics, wellness programs, and educational workshops, these facilities support the health and well-being of entire communities. This ensures that they receive clear instructions and understand their care plans, which is vital for managing chronic diseases.
Title Type Comments Close Key Information and Facilitating Understanding in Informed Consent Guidance for Sponsors, Investigators, and Institutional Review Boards Draft Guidance April 30 Early Alzheimer’s Disease: Developing Drugs for Treatment Draft Guidance May 13 Select Updates for the Premarket Cybersecurity Guidance: Section 524B of the (..)
Rare Pediatric Disease Priority Review Voucher Program Expiration Threat: The FDA’s popular voucher program for rare pediatric diseases is currently scheduled to expire as of September 30, 2024 unless it is renewed by Congress.
A wide range of other meetings that will be occurring this month, with various webinars, committee meetings, and workshops spanning a variety of topics. Upcoming Webinar Hosted by Xtalks Webinar/Seminar ( OPEN) Xtalks 11/6/2023 11/6/2023 What is the evidence for high-risk medical devices in the field of cardiovascular disease and diabetes?
Offer not valid on workshop only or academic/non-profit registrations. Register today. Offer applies to standard rates only and may not be combined with other offers, category rates, promotions or applied to an existing registration. The content of Sponsored Posts does not necessarily reflect the views of Pembroke Consulting, Inc.,
She is an American Institute for Medical and Biological Engineering (AIMBE) fellow and has co-chaired many international scientific conferences and workshops related to Chemical, Manufacturing and Controls (CMC) development strategies, including several co-sponsored by the U.S. Ma holds a PhD in chemical engineering from Yale University.
Of course, AA and P-line rats are not the only research animals bred for the study of disease. By doing so, researchers study pain, genetic disorders, the effect of drugs, and disease pathways in mammals that, while genetically similar to us, are not human beings. They are bred, incised, injected, and deprived.
Informa Connect’s Access USA March 19-21, 2024 | Pre-Conference Workshops on March 18, 2024 Philadelphia Marriott Downtown in Philadelphia, PA Exclusive Offer – Be sure to use your exclusive promocode 24AUSA10 to save 10% off* of your registration. What is Access USA?
Hands-on training course Webinar/ Seminar ( OPEN ) EMA 4/16/2024 4/16/2024 UKCA Sub-Group Committee Meeting ( CLOSED ) British In Vitro Diagnostics Association (BIVDA) 4/16/2024 4/17/2024 EMA multi-stakeholder workshop on psychedelics – Towards an E.U.
In addition to rare genetic diseases, talks at the Festival will address Crohn’s disease, rheumatoid arthritis, polycystic ovarian syndrome, and neurological disease and cancers. Workshops and panel discussions will address the power and challenges of single-cell transcriptomics and integrating multi-omics data.
Offer not valid on workshop only or academic/non-profit registrations. Drug Channels readers will save 10% off the current registration rate when they use code 21HUBDC10*. Offer applies to current rates only and may not be combined with other offers, category rates, promotions or applied to an existing registration.
Recognizing the issue of underrepresentation of racial and ethnic minorities in clinical trials, e.g., for Alzheimer’s Disease (AD), researchers have sought to understand the barriers and facilitators for patient access better. Although AD disproportionately affects U.S. Specifically, U.S. Rubin et al.
. “Johnson & Johnson is committed to supporting urgently-needed efforts to help identify the ‘missing millions’ of undiagnosed individuals with TB and connect them with care to help put the world back on track toward ending this disease by 2030.” TB is a forgotten respiratory disease that still kills 1.4
FDA offers a status check on its diversity in research provisions, one year post-FDORA At a workshop on FDA’s implementation of new statutory requirements for diversity in clinical research, agency and industry representatives gave a status update on implementation.
In addition to Deloitte and Evotec, S2S is also sponsored by Goodwin, JLL, USQ, and SVB, all of whom will be leading workshops to share their expertise with the presenters. These individuals will be mentoring the S2S Finalist presenters, along with mentors from the MassBio community.
This would facilitate certain minimal risk clinical investigations to support the development of new products to diagnose or treat diseases or conditions and would harmonize, to the extent practicable and consistent with statutory provisions, with the HHS Common Rule waiver provision that has been adopted and successfully employed by other agencies.
This will include a number of conferences, webinars, and training sessions, as well as a long-awaited EMA workshop on psychedelics. Perhaps the most significant pharmaceutical news anticipated for April: the vote on the pharmaceutical legislation revision. regulatory framework Webinar/ Seminar ( OPEN ) EMA 4/17/2024 4/17/2024 CEP 2.0:
July 1, 2023 MDUFA Finalize the draft guidance, “Content of Premarket Submissions for Device Software Functions,” by 18 months from close of the comment period.
The next town hall will focus on the clinical development of gene therapy products for rare diseases in February 2023. The workshop is also discussed in Cell & Gene here and here.
For example, on July 25, the Hungarian presidency hosted an informal meeting of health ministers to discuss health issues, such as mitigating the effect of cardiovascular diseases and implementing the European Health Data Space (EHDS).
RDDS 2023 focuses on collaboration – the foundation of success in rare disease research. You will meet individuals, advocacy leaders, industry, and research experts who have been there and done it, over two days of in-person only panel discussions, hands-on workshops, and expert office hours.”
Title Type Comments Close Fixed-Combinations and Single-Entity Versions of Previously Approved Antiretrovirals for the Treatment or Prevention of Human Immunodeficiency Virus-One Under the President’s Emergency Plan for Acquired Immunodeficiency Syndrome Relief Guidance November 1 Post-Warning Letter Meetings Under GDUFA Guidance November 6 In-Home (..)
Adding to all of this is the financial feasibility of generating small-batch CGT products for ultra-rare diseases with limited patient populations. Accelerated approval using surrogate endpoints is necessary to develop gene therapy products for rare disease populations. Current Regulatory Trends in Cell & Gene Therapy.
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