Critical Path Institute
Critical Path Institute
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The Influential Role of Patient Advocacy Groups in Registry Data Formats
RDCA-DAP's Metabolic Diseases Task Force presents, "The Infential Role of Patient Advocacy Groups in Registry Data Efforts." Led by Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-!) and Critical Path for Lysosomal Diseases Executive Director Amanda Klein, PharmD, this task force is dedicated to standardizing and integrating mitochondrial and inherited metabolic diseases data into RDCA-DAP, optimizing clinical trial data, and serving other unmet needs in inherited metabolic diseases.
About Critical Path Institute
C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org.
#CPath #drugdiscovery #researchgrants #globalhealth #drugdevelopment #datasharing #researchfunding #raredisease #academicresearch #regulatoryscience #globalhealth #consortia #collaboration #FDA #EMA #T1D #Parkinsons #Alzheimers #PKD #Duchenne #ALS #biomarkers
Переглядів: 62

Відео

Hidden in Plain Sight: Autosomal Dominant Tubulointerstitial Kidney Disease
Переглядів 608Місяць тому
Presented by Anthony Bleyer, MD, MS (Professor Nephrology at Wake Forest University School of Medicine) this presentation covered genetics of ADTKD, biology and mechanisms, Wake Forrest registry description, and unmet drug development needs. This webinar was brought to you by Critical Path Institute's Rare and Orphan Disease Program's Rare Disease Cures Accelerator-Data and Analytics Platform a...
I am BIO Podcast with Kristen Swingle, President & Chief Operating Officer Critical Path Institute
Переглядів 80Місяць тому
C-Path's President and COO Kristen Swingle sits down with I am BIO Podcast host Theresa Brady at the 2024 BIO Convention to discuss how biotech breakthroughs converge with solutions for patients and the planet. SUBSCRIBE to C-Path's UA-cam Channel Now ►► ua-cam.com/users/CriticalPathInstitute FOLLOW C-Path: ►LinkedIn: www.linkedin.com/company/c-path ►Facebook: CPathInstitute/ ►Twit...
Critical Path Institute Leads Collaborations
Переглядів 37Місяць тому
SUBSCRIBE to C-Path's UA-cam Channel Now ►► ua-cam.com/users/CriticalPathInstitute FOLLOW C-Path: ►LinkedIn: www.linkedin.com/company/c-path ►Facebook: CPathInstitute/ ►Twitter: CPathInstitute?re About Critical Path Institute C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path...
The Use and Development of DHTs with Patient Advocacy Groups
Переглядів 308Місяць тому
On Thursday, May 23, C-Path's RDCA-DAP team joined Dr. Laurent Servais of the University of Oxford and the University of Liège, for an overview of Actimyo and relevant learnings from use cases across rare disorders spanning different degrees of complexity and readiness. Additionally, patient advocacy group leaders will speak to their experience based on stage of development and the ability of A...
Webinar: TRxA BioBRIDGe Awards
Переглядів 1,1 тис.2 місяці тому
On Wednesday, May 15th, TRxA hosted a webinar to explain and review its 2024 funding opportunities. Content included an overview of the types of projects TRxA funds, eligibility criteria for BioBRIDGe awards, and details about how to apply before the first submission deadline of June 27, 2024. The webinar concluded with a live Q&A. SUBSCRIBE to C-Path's UA-cam Channel Now ►► ua-cam.com/users/Cr...
C-Path Roundtable: C-Path, Help4HD Unite to Discuss Advancements in Huntington's Disease Treatments
Переглядів 4692 місяці тому
Thomas Hart, Director of Outreach for Critical Path Institute’s Rare and Orphan Diseases program and program Executive Director Terina Martinez were joined by Jackson and Lauren Holder, Help4HD Radio’s producer and host, on Huntington's Disease Awareness Day. In the time that four spent together, the vigor, drive, compassion, and desire were on full display from four people who spend their live...
Clinical Outcome Assessment - Initial Public Discussion
Переглядів 1452 місяці тому
In this presentation from C-Path's rare and orphan disease program, you will see an overview of patient-focused drug development and endpoints that inform regulatory decision making, a summary of work to date on concepts and tools, as well as next steps for engaging with people with lived experience. SUBSCRIBE to C-Path's UA-cam Channel Now ►► ua-cam.com/users/CriticalPathInstitute FOLLOW C-Pat...
How Individuals and Organizations Can Support TRxA?
Переглядів 19 тис.2 місяці тому
Critical Path Institute's, C-Path, Translational Therapeutics Accelerator, or TRxA, is a game-changer in drug discovery and development. Its mission is simple yet significant: to provide academic researchers with funding and guidance for the advancement of novel therapeutics from the lab, through clinical trials, to commercialization, and ultimately to the patients who need them most. TRxA gran...
Improving Data Collection for Rare Epilepsies: Case example from the TSC Natural History Database
Переглядів 4854 місяці тому
Hosted by Critical Path Institute's Rare and Orphan Diseases program, this "Improving Data Collection for Rare Epilepsies" webinar welcomes speaker Elizabeth Cassidy, MPH, of TSC Alliance. Learn more at C-Path's Rare and Orphan Disease programs at: c-path.org/area-of-focus/rare-and-orphan-diseases/ #cpath #nonprofit #collaboration #epilepsy #raredisease
Rare Neurodegenerative Disease Efforts Under the ACT for ALS
Переглядів 6934 місяці тому
In this webinar from Critical Path Institute's Rare and Orphan Disease Programs, we discuss in-depth the how the ACT for ALS will affect those living with the disease. To learn more visit: c-path.org/cp-rnd #cpath #fda #collaboration #als #neuroscience #neurodenerativediseases
C-Path CEO Klaus Romero Guitar Shred for Show Your Stripes Campaign on Rare Disease Day 2024
Переглядів 1394 місяці тому
🦓 Rocking Out on #RareDiseaseDay! 🦓🎸 🌟 Get ready to rock and roll for a cause close to our hearts! 🤘 C-Path's very own CEO Klaus Romero is shredding in support of the National Organization for Rare Disorders' Show Your Stripes campaign for Rare Disease Day! Check out this electrifying video of our talented leader shredding on his Zebra guitar, all to raise awareness and support for those affect...
Critical Path Institute Expands Data Analytics to Mitochondrial and Inherited Metabolic Diseases
Переглядів 524 місяці тому
Critical Path Institute Expands Data Analytics to Mitochondrial and Inherited Metabolic Diseases at the Mitochondrial Medicine - Therapeutic Development Conference. This meeting is set to take place on the Wellcome Genome Campus in the UK (and virtually) from March 18-20. About Critical Path Institute Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-...
CIVM Workshop 2023 Day 2: Opening Remarks
Переглядів 444 місяці тому
Critical Path Institute's Complex In Vitro Model Workshop (CIVM) convened September 28-29, 2023. C-Path's Nick King and Graham Marsh kick off the mornnig. Learn more at c-path.org #cpath #collaboration #nonprofit #drugdevelopment #fda
CIVM 2023 Day 2 : Breakout Sessions - Drafting a Qualification Utilizing Sessions 1, 2, and 3
Переглядів 565 місяців тому
Critical Path Institute's Complex In Vitro Model Workshop (CIVM) convened September 28-29, 2023. C-Path's Klaus Romero, Graham Marsh, and Nick King led breakout sessions with attendees to draft a qualification outline. Learn more at c-path.org #cpath #collaboration #nonprofit #drugdevelopment #fda
CIVM Workshop Day One: Perspective on Translating New Drug Development Tools into Regulatory Use
Переглядів 385 місяців тому
CIVM Workshop Day One: Perspective on Translating New Drug Development Tools into Regulatory Use
SBS Day 2 - Session IV: Clinical Trial Design Considerations
Переглядів 525 місяців тому
SBS Day 2 - Session IV: Clinical Trial Design Considerations
SBS Day 2 - Panel IV-b Considerations for Clinical Trial Design Optimization in Neonates and T1D
Переглядів 435 місяців тому
SBS Day 2 - Panel IV-b Considerations for Clinical Trial Design Optimization in Neonates and T1D
SBS Day 2 - Session III-b: Disease Prevention in Lysosomal Diseases
Переглядів 635 місяців тому
SBS Day 2 - Session III-b: Disease Prevention in Lysosomal Diseases
SBS Day 2 - Disease Prevention in T1D and Neonates Panel
Переглядів 735 місяців тому
SBS Day 2 - Disease Prevention in T1D and Neonates Panel
SBS Day 2 - INC current perspective: Conducting gene therapy clinical trials
Переглядів 585 місяців тому
SBS Day 2 - INC current perspective: Conducting gene therapy clinical trials
SBS Day 2 - Session III-A Disease Prevention in T1D and Neonates (Case Study)
Переглядів 695 місяців тому
SBS Day 2 - Session III-A Disease Prevention in T1D and Neonates (Case Study)
SBS Day 2 - Session III Disease Prevention
Переглядів 645 місяців тому
SBS Day 2 - Session III Disease Prevention
Understanding Disease Progression Models: What are They, Why are They Useful, & How Are They Applied
Переглядів 4535 місяців тому
Understanding Disease Progression Models: What are They, Why are They Useful, & How Are They Applied
Webinar: TRxA 2024 Funding Opportunities
Переглядів 2235 місяців тому
Webinar: TRxA 2024 Funding Opportunities
CIVM Workshop 2023 Day 2: A Clinical Reviewer's Perspective on CIVM for Drug Development
Переглядів 386 місяців тому
CIVM Workshop 2023 Day 2: A Clinical Reviewer's Perspective on CIVM for Drug Development
CIVM Workshop 2023 Day 1: Identifying Appropriate CIVM for a COU
Переглядів 876 місяців тому
CIVM Workshop 2023 Day 1: Identifying Appropriate CIVM for a COU
CIVM Workshop 2023 Day 2: Reflecting on Refined Contexts of Use
Переглядів 256 місяців тому
CIVM Workshop 2023 Day 2: Reflecting on Refined Contexts of Use
CIVM Workshop 2023 Day 1: Biological Performance Considerations Panel Discussion
Переглядів 356 місяців тому
CIVM Workshop 2023 Day 1: Biological Performance Considerations Panel Discussion
CIVM Workshop 2023 Day 1: Biological Performance Considerations
Переглядів 1276 місяців тому
CIVM Workshop 2023 Day 1: Biological Performance Considerations

КОМЕНТАРІ

  • @sward007
    @sward007 4 місяці тому

    Ah, very cool Klaus. Learn something new every day!

  • @paulhetherington3854
    @paulhetherington3854 6 місяців тому

    TRX = Jap women - vehicle CO.! You totally - got caught in - hindu faith practice!

  • @henryw8385
    @henryw8385 7 місяців тому

    👊 Promo>SM

  • @olazai4440
    @olazai4440 9 місяців тому

    Thank you 🙏

  • @Humayrarannaghor1
    @Humayrarannaghor1 Рік тому

    Very good video

  • @VaidyanathanNatarajan
    @VaidyanathanNatarajan Рік тому

    Radiation effects on genetic mutations is the key to stop new diseases. ""One child four years back has become ten now"". Many data's on radio wave (cell phones), effect on human (not just cancer), specifically on sperm and during child in mothers womb. They are specifically hidden in silos of mobile r&d labs. Hope people will come out and take preventive steps. Good LUCK.

  • @cristianreyes7742
    @cristianreyes7742 Рік тому

    Oms should be Sue for not preventing pregnant women about this

  • @itlsacademy
    @itlsacademy Рік тому

    Can you help me out, this this is diagnosed in my child, his age is 9.5 years

  • @benken9601
    @benken9601 Рік тому

    Hello everyone am very happy today am cure totally completely forever form herpes simplex disease with the help of Dr omoye you can also contact him on his UA-cam channel ua-cam.com/channels/lshMWAD9EFaKUTbdbtBH7A.html He also cure all kind disease Herpes simplex 1&2 Diabetes medication Penis enlargement product STD infection medication Monkeypox He also cure all kinds of disease such as Herpes simplex 1&2 Diabetes medication Penis enlargement product STD infection medication Monkeypox HIV/AIDS Low sperm can Erectile dysfunction Diabetes Hepatitis Pregnancy product High blood pressure Low sugar level High sugar level Infections Body odor🌿👈🏻

  • @kirkbowett5685
    @kirkbowett5685 Рік тому

    I appreciate your determination in showing me I can be healed from the Herpes simplex virus, Thanking you for your perseverance, support, and love, thank you Dr Ofenmu UA-cam channel for being the wonderful doctor..

  • @freepongquelini.loanfirm6309

    This has been a challenging time, and I appreciate you so much Dr Ofenmu UA-cam channel , You have created a life that I thought was lost, thank you so much grateful my sister's genital warts I am grateful.

  • @mrsam3236
    @mrsam3236 Рік тому

    did any body produce it in INDIA ,IF SO PLEASE wtsaap me

  • @acunamarlen5358
    @acunamarlen5358 2 роки тому

    Herbal treatment is 100% guarantee HSV 1&2 cure, the reason why most people are finding it difficult to cure HSV is because they believe on medical report, drugs medical treatment which is not helpful to cure HSV. Natural roots/herbs are the best remedy which can easily eradicate herpes cure forever, from information about herpes cure Please write #drabiolaefe Toutube

  • @nickkane4645
    @nickkane4645 2 роки тому

    I know the suffering that comes with herpes, disturbing day and night thank God I got rid of the virus permanently with Dr Oseghale Sunday Herbal Mixture I am really happy that I'm safe now get yours also, be safe. #DrOseghaleSundayHerbalHome

  • @paulmaguire2683
    @paulmaguire2683 2 роки тому

    Excellent overview - thanks

  • @mrmarkjacksonnt8806
    @mrmarkjacksonnt8806 2 роки тому

    Meeting you Dr uma on You Tube is like meeting GOD himself because you deed in my life what no doctor can't do. Thanks you for curing my herpes virus completely you are a life saver, you can also contact him on youtube #drumaherbalhome

  • @danielacora1995
    @danielacora1995 2 роки тому

    All thanks to #drobudu for curing I and my wife from Herpes, Gratitude Sir

  • @smithlusi9415
    @smithlusi9415 2 роки тому

    Meeting you Dr uma on You Tube is like meeting GOD himself because you deed in my life what no doctor can't do. Thanks you for curing my herpes virus completely you are a life saver, you can also contact him on youtube #drumaherbalhome

  • @francesshud474
    @francesshud474 2 роки тому

    Meeting you Dr uma on You Tube is like meeting GOD himself because you deed in my life what no doctor can't do. Thanks you for curing my herpes virus completely you are a life saver, you can also contact him on youtube #drumaherbalhome

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Following

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Plz make cure or atleast avoid dialysis and transplant treatment for pkd patients. We hope from you. We prayer for you may god bless you

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    I have polycystics kidney disease. I sign up on c-path. But I am not getting reply. Kindly look at us we suffering rare diseaes thanks

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    May are they going therapy by drug for rare kidney disease?

  • @rubyabrams243
    @rubyabrams243 2 роки тому

    Exciting times ahead!

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Plz bring soon therapy for adpkd in markete we whole families suffering from adpkd thanks

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Following

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Following

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Following

  • @adeelmehmood8093
    @adeelmehmood8093 2 роки тому

    Following

  • @janegunther1785
    @janegunther1785 2 роки тому

    Very articulate, Justin! I am interested in the RDCA-DAP and your insights were helpful to understanding what the platform can do. I think you will have no obstacles to finding employment when you have completed your academic studies.

  • @nwc3270
    @nwc3270 2 роки тому

    Sernova Corp is having great results with the trial they have going right now.

  • @luiscarrillo1322
    @luiscarrillo1322 2 роки тому

    Hi, 10 MG of Propanolol Is the dosis for a adult person? My father have 82 yrs and have angiosarcoma in skin on the face.

  • @katrecemiller8325
    @katrecemiller8325 2 роки тому

    Herpes is a common infection/condition, affecting about 1 in 4 American adults. Hsv1 & Hsv2 are the common types of Herpes virus and if untreated, they can get unbearable and cause severe medical issues. I'm so glad I'm over herpes and its stigma, all thanks to Dr. Aloha ua-cam.com/channels/_YFEEZEr1BxGkNg1d4vqww.html ☘️🇺🇸☘️🇺🇸

  • @jessicaroland8921
    @jessicaroland8921 2 роки тому

    I never believed that there is a permanent cure for herpes virus until I applied natural herbal supplements from Dr Erayo on UA-cam and I was completely cured in less than a month. 😘💜🌿

  • @jessicaroland8921
    @jessicaroland8921 2 роки тому

    I never believed that there is a permanent cure for herpes virus until I applied natural herbal supplements from Dr Erayo on UA-cam and I was completely cured in less than a month. 😘💜🌿

  • @aishahabibu1184
    @aishahabibu1184 2 роки тому

    Glory be to God for using Dr Alieaotor on UA-cam to cure my HSV with pure natural herbal supplements 🍀🥰💙🍀

  • @aishahabibu1184
    @aishahabibu1184 2 роки тому

    Glory be to God for using Dr Alieaotor on UA-cam to cure my HSV with pure natural herbal supplements 🍀🥰💙🍀

  • @MetadataCenterOrg
    @MetadataCenterOrg 2 роки тому

    Thank you! A really nice job describing an important and complex subject.

  • @sandrasermone140
    @sandrasermone140 2 роки тому

    I hope that you all will start working with ADNP syndrome

  • @ullajenny5820
    @ullajenny5820 2 роки тому

    Glory to God i won herpes #Doctorojie herbs cured my herpes Praise master Jesus

  • @ullajenny5820
    @ullajenny5820 2 роки тому

    Glory to God i won herpes #Doctorojie herbs cured my herpes ....,

  • @user-kd7zc8dt2z
    @user-kd7zc8dt2z 2 роки тому

    7rkwti #von.ong

  • @HealthTips2M
    @HealthTips2M 2 роки тому

    Nice 👍👍 hame bhi saport kare plases 🙏

  • @fernandocharlton5063
    @fernandocharlton5063 2 роки тому

    zfzjm #vur.fyi

  • @Jerseyboondocks
    @Jerseyboondocks 3 роки тому

    I just found out my GFR is 57...it was just 99 last year. I am only 44 and it is rapidly progressing. I am very sad and in pain from terrible big kidneys and dangerous high blood pressure. I was never given tolvapton and now is it too late for it to help me ?? Please help answer thank you so much

  • @mussiekebede2575
    @mussiekebede2575 3 роки тому

    Nice Presentation, guys!

  • @mykedoes4099
    @mykedoes4099 3 роки тому

    Evidence of a genetic pre disposition is evidence of parasitic infection , because it is a known fact that parasites manipulate your genetics. Case in point

  • @mykedoes4099
    @mykedoes4099 3 роки тому

    Since half of all children are born with Lyme it is not far fetched theory i propose the medical community recognize.

  • @mykedoes4099
    @mykedoes4099 3 роки тому

    Someone in the medical community should consider the fact that parasites can change your dna so that they may live inside you , hence genetic diseases like PKD "a pkd victim who believes his condition is caused by the Lyme parasite" parasites create biofilm which is awfully similar if not identical to a cyst .

  • @krystnkuckelman8011
    @krystnkuckelman8011 3 роки тому

    Jaina is so brave. Thank you for sharing your story.