Канал: Orphan Drugs

World Orphan Drug Congress Europe 2024 Trailer

World Orphan Drug Congress Europe 2024 Trailer

World Orphan Drug Congress Europe 2023 Highlights

World Orphan Drug Congress Europe 2023 Highlights

World Orphan Drug Congress Europe | Rare Disease Day 2024 - Exclusive Patient Spotlight

World Orphan Drug Congress Europe | Rare Disease Day 2024 - Exclusive Patient Spotlight

World Orphan Drug Congress Europe | Rare Disease Day 2024

World Orphan Drug Congress Europe | Rare Disease Day 2024

World Orphan Drug Congress 2023 Trailer

World Orphan Drug Congress 2023 Trailer

World Orphan Drug Congress 2020

World Orphan Drug Congress 2020

World Orphan Drug Congress 2018, 6-8 November, Barcelona

World Orphan Drug Congress 2018, 6-8 November, Barcelona

World Orphan Drug Congress 2018 Announcement

World Orphan Drug Congress 2018 Announcement

Chris Garabedian - Orphan Drug Congress Interview Part 7

Chris Garabedian - Orphan Drug Congress Interview Part 7

Chris Garabedian - Orphan Drug Congress Interview Part 6

Chris Garabedian - Orphan Drug Congress Interview Part 6

Chris Garabedian - Orphan Drug Congress Interview Part 5

Chris Garabedian - Orphan Drug Congress Interview Part 5

Chris Garabedian - Orphan Drug Congress Interview Part 4

Chris Garabedian - Orphan Drug Congress Interview Part 4

Chris Garabedian - Orphan Drug Congress Interview Part 3

Chris Garabedian - Orphan Drug Congress Interview Part 3

Chris Garabedian - Orphan Drug Congress Interview Part 2

Chris Garabedian - Orphan Drug Congress Interview Part 2

Chris Garabedian - Orphan Drug Congress Interview Part 1

Chris Garabedian - Orphan Drug Congress Interview Part 1

Speed Networking Video

Speed Networking Video

Martin Andrews Senior Vice President, Rare Disease, GSK Part 1

Martin Andrews Senior Vice President, Rare Disease, GSK Part 1

Martin Andrews Senior Vice President, Rare Disease, GSK Part 2

Martin Andrews Senior Vice President, Rare Disease, GSK Part 2

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial Feedback

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial Feedback

Martin Andrews Senior Vice President, Rare Disease, GSK Part 2

Martin Andrews Senior Vice President, Rare Disease, GSK Part 2

Dr Segolene Ayme Emeritus Research Director at INSERM, Project Leader of IRDiRC SUPPORT

Dr Segolene Ayme Emeritus Research Director at INSERM, Project Leader of IRDiRC SUPPORT

Dr Thomas Meier CEO, Santhera Pharmaceuticals Holding Ltd

Dr Thomas Meier CEO, Santhera Pharmaceuticals Holding Ltd

Mark Corbett Chief Operating Officer, Clinigen Group Part 2

Mark Corbett Chief Operating Officer, Clinigen Group Part 2

Katherine K Leon Co Founder and Director of Communications, SCAD Aliliance Part 2

Katherine K Leon Co Founder and Director of Communications, SCAD Aliliance Part 2

Dr Derralynn Hughes Senior Lecturer Honorary Consultant, Royal Free London NHS Foundation Trusts

Dr Derralynn Hughes Senior Lecturer Honorary Consultant, Royal Free London NHS Foundation Trusts

Richard Allcorn Co Founder, Comradis

Richard Allcorn Co Founder, Comradis

H Termeer Speech Final

H Termeer Speech Final

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group

Jutta Roth Business Development Manager, University of Oxford

Jutta Roth Business Development Manager, University of Oxford

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial

Prof Paul Lasko Chair, IRDiRC

Prof Paul Lasko Chair, IRDiRC

Martin Andrews Senior Vice President, Rare Disease, GSK Part 1

Martin Andrews Senior Vice President, Rare Disease, GSK Part 1

Prof Paul Lasko Chair, IRDiRC Testimonial

Prof Paul Lasko Chair, IRDiRC Testimonial

Mark Corbett Chief Operating Officer, Clinigen Group Part 1

Mark Corbett Chief Operating Officer, Clinigen Group Part 1

Mark Rothera Chief Commercial Officer, PTC Therapeutics

Mark Rothera Chief Commercial Officer, PTC Therapeutics

Eibhlin Mulroe CEO, IPPOSI

Eibhlin Mulroe CEO, IPPOSI

Dr Segolene Ayme Emeritus Research Director at INSERM, Project Leader of IRDiRC SUPPORT

Dr Segolene Ayme Emeritus Research Director at INSERM, Project Leader of IRDiRC SUPPORT

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial

Sue Routledge Co Manager of the Pitt Hopkins Syndrome Support Group Testimonial

Katherine K Leon Co Founder and Director of Communications, SCAD Aliliance Part 1

Katherine K Leon Co Founder and Director of Communications, SCAD Aliliance Part 1

Dr Jonathan Morton Communications Director Rare and Orphan Diseases Practice , Oxford PharmaGenesi

Dr Jonathan Morton Communications Director Rare and Orphan Diseases Practice , Oxford PharmaGenesi

Henri Termeer at WODC USA 2015 - Teaser Video

Henri Termeer at WODC USA 2015 - Teaser Video

Total Orphan Drugs #ALSIceBucketChallenge

Total Orphan Drugs #ALSIceBucketChallenge

#ALSIceBucketChallenge- Pinky WODC USA

#ALSIceBucketChallenge- Pinky WODC USA

Finding children with rare disease during their “window of opportunity”

Finding children with rare disease during their “window of opportunity”

Costly ticket, worth entry – why Brazil should be on every orphan drug developer’s radar

Costly ticket, worth entry – why Brazil should be on every orphan drug developer’s radar

Panel session Orphan drug licensing and M&A trends Learnings from 2013 and what to expect in 2014

Panel session Orphan drug licensing and M&A trends Learnings from 2013 and what to expect in 2014

Myths and realities of the Canadian orphan drug market

Myths and realities of the Canadian orphan drug market

Early access to orphan drugs – the rationale, challenges and options available

Early access to orphan drugs – the rationale, challenges and options available

Outsourcing in Rare Disease - obtaining an accurate bid from a CRO

Outsourcing in Rare Disease - obtaining an accurate bid from a CRO

A robust and functioning system to evaluate and consider the benefit-risk of a potential therapy

A robust and functioning system to evaluate and consider the benefit-risk of a potential therapy

What’s NCATS role in increasing the speed of translating research to rare disease therapies

What’s NCATS role in increasing the speed of translating research to rare disease therapies

The downstream of genomic sequencing - bridging the gap from diagnosis to therapies

The downstream of genomic sequencing - bridging the gap from diagnosis to therapies

Balancing innovation, accessibility, and unsustainable costs

Balancing innovation, accessibility, and unsustainable costs

Regulatory considerations for the development of substantial evidence of effectiveness

Regulatory considerations for the development of substantial evidence of effectiveness

Serious medical conditions: strategic US regulatory considerations for drug development

Serious medical conditions: strategic US regulatory considerations for drug development

Rare disease collaboration model - big pharma embraces a new approach to drug development

Rare disease collaboration model - big pharma embraces a new approach to drug development

Interview: Siren Interactive's Wendy White at the WODC USA 2014

Interview: Siren Interactive's Wendy White at the WODC USA 2014

Interview: Quintiles' Eric Gemmen at the WODC USA 2014

Interview: Quintiles' Eric Gemmen at the WODC USA 2014

Interview: ProMetic's Pierre Laurin at the WODC USA 2014

Interview: ProMetic's Pierre Laurin at the WODC USA 2014

Interview: Neonc Technologies' Thomas Chen at the WODC USA 2014

Interview: Neonc Technologies' Thomas Chen at the WODC USA 2014

Interview: NanoBlood's Carleton J.C. Hsia at the WODC USA 2014

Interview: NanoBlood's Carleton J.C. Hsia at the WODC USA 2014

Interview: University of Minnesota's Ramaiah Muthyala at the WODC USA 2014

Interview: University of Minnesota's Ramaiah Muthyala at the WODC USA 2014

Interview: Humana's Roy Beveridge at the WODC USA 2014

Interview: Humana's Roy Beveridge at the WODC USA 2014

Interview: Hansa Medical's Emanuel Björne at the WODC USA 2014

Interview: Hansa Medical's Emanuel Björne at the WODC USA 2014

World Orphan Drug Congress Europe 2013

World Orphan Drug Congress Europe 2013

Leveraging protocol-driven natural history studies into clinical development plans and registration

Leveraging protocol-driven natural history studies into clinical development plans and registration

A New Model for Drug Development in Rare Disease: Gaining Approval as a Class / Chris Garabedian, Pr

A New Model for Drug Development in Rare Disease: Gaining Approval as a Class / Chris Garabedian, Pr

Orphan Drugs -- small market/big opportunity / Craig Kephart, President , Centric Health Resources

Orphan Drugs -- small market/big opportunity / Craig Kephart, President , Centric Health Resources

From large to small: the critical elements of orphan drug success / David Meeker, COO, Genzyme

From large to small: the critical elements of orphan drug success / David Meeker, COO, Genzyme

Accelerating Drug Discovery and Advancing Knowledge in Huntington's disease through the use of CRO

Accelerating Drug Discovery and Advancing Knowledge in Huntington's disease through the use of CRO

The growing role of patient groups taking charge of all parts of the value chain / Frédéric Revah,

The growing role of patient groups taking charge of all parts of the value chain / Frédéric Revah,

Entering into the clinic with an intracerebral gene therapy product / Karen Aiach, Project Manager,

Entering into the clinic with an intracerebral gene therapy product / Karen Aiach, Project Manager,

Achieving the clinical added value required for approval / Hans Schikan, CEO, Prosensa

Achieving the clinical added value required for approval / Hans Schikan, CEO, Prosensa

Case study: Challenges of ultra-orphan drug development: Glybera, a gene therapy for LPL deficiency

Case study: Challenges of ultra-orphan drug development: Glybera, a gene therapy for LPL deficiency

Patient registries among the range of data sources and methodologies available for orphan drugs / Na

Patient registries among the range of data sources and methodologies available for orphan drugs / Na

Developing a patient registry into an online rare disease community?/ Dr Nick Sireau, Chairman, AKU

Developing a patient registry into an online rare disease community?/ Dr Nick Sireau, Chairman, AKU

Developing a patient registry into an online rare disease community? / Dr Nick Sireau, Chairman, AKU

Developing a patient registry into an online rare disease community? / Dr Nick Sireau, Chairman, AKU

Natural History Registries (Patient Registries) / Vanesa Rangel Miller, Genetic Counselor, Patient C

Natural History Registries (Patient Registries) / Vanesa Rangel Miller, Genetic Counselor, Patient C

Engaging in an open dialogue throughout the clinical trial stage / Annie-Claude Benichou, Alaxia / T

Engaging in an open dialogue throughout the clinical trial stage / Annie-Claude Benichou, Alaxia / T

From big pharma to niche specialist: lessons learned from rare disease collaborations / Dr. Alvin S

From big pharma to niche specialist: lessons learned from rare disease collaborations / Dr. Alvin S

Keeping up with the times: social media in the orphan drug industry / Robert Derham, Founder, CheckO

Keeping up with the times: social media in the orphan drug industry / Robert Derham, Founder, CheckO

Developing a patient registry into an online rare disease community? / Samantha Parker, Orphan Europ

Developing a patient registry into an online rare disease community? / Samantha Parker, Orphan Europ

The ultimate data capture: creating a global registry of patient registries / Sharon Terry, CEO, Gen

The ultimate data capture: creating a global registry of patient registries / Sharon Terry, CEO, Gen

When a proof-of-concept study turns into a pivotal trial: Experiences with Leber's Hereditary Opti

When a proof-of-concept study turns into a pivotal trial: Experiences with Leber's Hereditary Opti

A frank evaluation of the value of an orphan drug treatment / Segolene Ayme, Director of Research, I

A frank evaluation of the value of an orphan drug treatment / Segolene Ayme, Director of Research, I

Developing new HTA methods to evaluate rare disease treatments / Ken O'Reilly, Director of Patient A

Developing new HTA methods to evaluate rare disease treatments / Ken O'Reilly, Director of Patient A

Building the strategy to diagnose all and treat 200 rare diseases / Catherine Berens, Scientific Of

Building the strategy to diagnose all and treat 200 rare diseases / Catherine Berens, Scientific Of

Addressing global health issues: the inverse of orphan drug development / Jerry Zeldis, CMO, Celgene

Addressing global health issues: the inverse of orphan drug development / Jerry Zeldis, CMO, Celgene

The decision-making process for ultra-orphan drug availability in England / Josie Godfrey, Head of P

The decision-making process for ultra-orphan drug availability in England / Josie Godfrey, Head of P

Early access to orphan drugs -- the rationale, challenges and options available / Mark Corbett, Glo

Early access to orphan drugs -- the rationale, challenges and options available / Mark Corbett, Glo

Expanding product indications through increased scientific understanding / Peter Myrenfors, Director

Expanding product indications through increased scientific understanding / Peter Myrenfors, Director

Creating a patient-orientated environment through the National Rare Disease Plans / Segolene Ayme, D

Creating a patient-orientated environment through the National Rare Disease Plans / Segolene Ayme, D

The ultimate beneficiary or the forgotten endpoint: a patient organisation perspective / Sue Millman

The ultimate beneficiary or the forgotten endpoint: a patient organisation perspective / Sue Millman

Collaborative innovation: the international ambitions of the IRDiRC / Sharon Terry, CEO, Genetic All

Collaborative innovation: the international ambitions of the IRDiRC / Sharon Terry, CEO, Genetic All

Strategies for ensuring reimbursement and improving patient access / Carlo Incerti, SVP Global Medic

Strategies for ensuring reimbursement and improving patient access / Carlo Incerti, SVP Global Medic

Company profile / Lyn Wiesinger, Swedish Orphan Biovitrum, World Orphan Drug Congress USA

World Orphan Drug Congress USA 2011 event highlights - North America's leading orphan drug conferenc

World Orphan Drug Congress USA 2011 event highlights - North America's leading orphan drug conferenc

A recipe for commercial success in orphan disease Pt 2 - Daniel Maher, BioMarin

A recipe for commercial success in orphan disease Pt 2 - Daniel Maher, BioMarin

A recipe for commercial success in orphan disease - Daniel Maher, BioMarin

A recipe for commercial success in orphan disease - Daniel Maher, BioMarin

Swedish Orphan Biovitrum - Lyn Wiesinger, Biovitrum - World Orphan Drug Congress USA 2011

Swedish Orphan Biovitrum - Lyn Wiesinger, Biovitrum - World Orphan Drug Congress USA 2011

Orphan drug landscape in Japan Pt 2 - Nobuo Uemura, Pharma and Medical Devices Agency Japan

Orphan drug landscape in Japan Pt 2 - Nobuo Uemura, Pharma and Medical Devices Agency Japan

Orphan drug landscape in Japan - Nobuo Uemura, Pharma and Medical Devices Agency Japan

Orphan drug landscape in Japan - Nobuo Uemura, Pharma and Medical Devices Agency Japan

How to approach orphan and genetic diseases Pt 2 - Dr Ed Masciolo, Pfizer

How to approach orphan and genetic diseases Pt 2 - Dr Ed Masciolo, Pfizer

How to approach orphan and genetic diseases - Dr Ed Masciolo, Pfizer

How to approach orphan and genetic diseases - Dr Ed Masciolo, Pfizer

Patient testimony - John W. Walsh, Apha -1 Foundation - World Orphan Drug Congress USA 2011

Patient testimony - John W. Walsh, Apha -1 Foundation - World Orphan Drug Congress USA 2011

Genzyme's patient-focused approach - John Butler, Genzyme - World Orphan Drug Congress USA 2011

Genzyme's patient-focused approach - John Butler, Genzyme - World Orphan Drug Congress USA 2011

Orphan drugs and rare diseases: pharma executives at the World Orphan Drug Congress USA 2011

Orphan drugs and rare diseases: pharma executives at the World Orphan Drug Congress USA 2011