Rachel Yang | Senior Advisor
Zhongshan Hospital Fudan University

Day 1 - Wednesday 23 October @ 14:10

Convergence on rare disease and precision medicine – a real drive on drug development

Moderator: Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International

×

Durhane Wong-Rieger / Convergence on rare disease and precision medicine – a real drive on drug development

Durhane Wong-Rieger, President, Chief Executive Officer,
Canadian Organisation for Rare Disorder & Rare Diseases International

DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...

CloseMore

Federica Miotto, Translational Project Manager, Fondazione Telethon

Denis Costello, Director, CML Advocates Network

Sonia VALERO, Head Of Clinical Operations - Patient Advocacy Leader, Vivet Therapeutics

×

Sonia VALERO / Convergence on rare disease and precision medicine – a real drive on drug development

Sonia VALERO, Head Of Clinical Operations - Patient Advocacy Leader,
Vivet Therapeutics

Sonia Valero, Head of Clinical Operations and Patient Advocacy representative, Vivet Therapeutics Pharmacist by training with a specialization in neurosciences, Sonia Valero has over 25 years of experience in drug development across early to late phases in CRO, pharma, and biotech. She has...

CloseMore

Rachel Yang, Senior Advisor, Zhongshan Hospital Fudan University

×

Rachel Yang / Convergence on rare disease and precision medicine – a real drive on drug development

Rachel Yang, Senior Advisor,
Zhongshan Hospital Fudan University

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

CloseMore

Maciek Klein, Global Chief Business Officer, Saventic Health GmbH

×

Maciek Klein / Convergence on rare disease and precision medicine – a real drive on drug development

Maciek Klein, Global Chief Business Officer,
Saventic Health GmbH

Maciek Klein is the Global Chief Business Officer at Saventic Health, a MedTech start-up leveraging Artificial Intelligence to screen electronic health records and identify patients with rare and hidden diseases. With a background in Economics and Business, Maciek leads customer acquisition and...

CloseMore

Day 2 - Thursday 24 October @ 11:10

Panel: Accelerating rare disease diagnosis beyond birth

-Genetic newborn screening and digital technologies

Moderator: Colin O'Donnell, Head of EUCAN & International Government Affairs, Alexion – AstraZeneca Rare Disease

×

Colin O'Donnell / Panel: Accelerating rare disease diagnosis beyond birth

Colin O'Donnell, Head of EUCAN & International Government Affairs,
Alexion – AstraZeneca Rare Disease

Colin has lead EUCAN & International Government Affairs for Alexion, AstraZeneca’s Rare Disease Unit, since May 2023, responsible for creating international government affairs strategy and executing global policy strategy in rare diseases, while creating opportunities for prioritization of rare ...

CloseMore

Julian Isla, Director, Foundation 29

×

Julian Isla / Panel: Accelerating rare disease diagnosis beyond birth

Julian Isla, Director,
Foundation 29

Julian Isla is the founder of the European Dravet Syndrome Federation, an European organization of patients with Dravet Syndrome. Julian Isla founded Dravet Syndrome Foundation twelve years ago. Dravet Syndrome Foundation is committed to find new treatment for Dravet Syndrome, an epileptic...

CloseMore

Stefano Portolano, CEO, Azafaros

×

Stefano Portolano / Panel: Accelerating rare disease diagnosis beyond birth

Stefano Portolano, CEO,
Azafaros

Stefano Portolano joined Azafaros in 2021, bringing with him 20+ years in orphan disease and speciality drugs and a remarkable track record in building and developing high-performing teams. He successfully led numerous commercial, market access and medical affairs efforts in Europe and the...

CloseMore

Rachel Yang, Senior Advisor, Zhongshan Hospital Fudan University

×

Rachel Yang / Panel: Accelerating rare disease diagnosis beyond birth

Rachel Yang, Senior Advisor,
Zhongshan Hospital Fudan University

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

CloseMore

Maurizio Scarpa, Director, Coordinating Center For Rare Diseases, MetabERN

Session led by: alexion-–-astrazeneca-rare-disease

Day 2 - Thursday 24 October @ 14:10

Panel: From global to local: partnering for patient centered policies beyond Europe

The panel aims to highlight the synergies that can be developed between the global and local levels to support policy shaping that addresses the needs of the rare disease communities and help identify what are key success factors to drive policy change locally and translate global commitments into impactful actions for PLWRD.

The panel will explore what is currently existing and what is on the horizon for rare diseases globally, and invite panellists from China, Spain, the UAE, Canada, and Brazil, as well as the audience, to share examples and reflect on how these global frameworks can be leveraged to create traction nationally and locally. The panel will also explore how we can build on local good practices existing “on the ground” within different healthcare models.

Moderator: Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International

×

Durhane Wong-Rieger / Panel: From global to local: partnering for patient centered policies beyond Europe

Durhane Wong-Rieger, President, Chief Executive Officer,
Canadian Organisation for Rare Disorder & Rare Diseases International

DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...

CloseMore

Antoine Daher, President, Casa Hunter

Roser Francisco, Head of the Clinical Accreditation Unit, Hospital Sant Joan de Déu

×

Roser Francisco / Panel: From global to local: partnering for patient centered policies beyond Europe

Roser Francisco, Head of the Clinical Accreditation Unit,
Hospital Sant Joan de Déu

PhD qualified with 20+ years of experience in Basic Research, Clinical Trial Monitoring, Patient Registry design and monitoring, Healthcare Policies design and implementation, Multidisciplinary Reference Units accreditation and monitoring, Patient Advocacy and Bioethics, within an academic Health...

CloseMore

Rachel Yang, Senior Advisor, Zhongshan Hospital Fudan University

×

Rachel Yang / Panel: From global to local: partnering for patient centered policies beyond Europe

Rachel Yang, Senior Advisor,
Zhongshan Hospital Fudan University

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

CloseMore

Flaminia Macchia, Head, Global Rare Disease Policy, Alexion – AstraZeneca Rare Disease

Session led by: alexion-–-astrazeneca-rare-disease

Day 2 - Thursday 24 October @ 15:25

Panel: Creating Pathways to meet opportunities and challenges of ultra-rare diseases

Moderator: Durhane Wong-Rieger, President, Chief Executive Officer, Canadian Organisation for Rare Disorder & Rare Diseases International

×

Durhane Wong-Rieger / Panel: Creating Pathways to meet opportunities and challenges of ultra-rare diseases

Durhane Wong-Rieger, President, Chief Executive Officer,
Canadian Organisation for Rare Disorder & Rare Diseases International

DURHANE WONG-RIEGER, PHD is Chair of Rare Disease International, President of Asia Pacific Alliance of Rare Disease Organizations, Treasurer of UN NGO for Rare Diseases, Chair of Patient Advocates Constituency Committee of International Rare Disease Research Consortium, and on the Editorial Boards...

CloseMore

Parvathy Krishnan, Executive Director, Krishnan Family Foundation

×

Parvathy Krishnan / Panel: Creating Pathways to meet opportunities and challenges of ultra-rare diseases

Parvathy Krishnan, Executive Director,
Krishnan Family Foundation

Parvathy is a Rare Disease Specialist and healthcare professional with a Masters in Clinical Nutrition. Parvathy is an expert in Patient Advocacy and Patient caregiver education. Parvathy has dedicated her life to empowering people worldwide to advocate for themselves no matter their location or...

CloseMore

Rachel Yang, Senior Advisor, Zhongshan Hospital Fudan University

×

Rachel Yang / Panel: Creating Pathways to meet opportunities and challenges of ultra-rare diseases

Rachel Yang, Senior Advisor,
Zhongshan Hospital Fudan University

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

CloseMore

Paolo Morgese, Vice President, Public Affairs, Europe, Alliance for Regenerative Medicine

Flaminia Macchia, Head, Global Rare Disease Policy, Alexion – AstraZeneca Rare Disease

Day 2 - Thursday 24 October @ 16:30

Keynote: How do we reinvent the healthcare system to meet the demand of not only rare diseases, but potentially all patients in future?

-By 2025 we will have many more gene therapies/ATMPs in development/approved. With this future healthcare systems cannot have bespoke solutions for each new ATMP, how can industry work with the healthcare system to build models of care that are effective and sustainable

-How can industry create solutions facilitate patient access to gene therapies?

-How can companies and the healthcare system work together to make it more equitable?

-What role can clinicians and patients play in developing a system that is efficient and delivers the outcomes expected?

Moderator: Sheela Upadhyaya, Life Science Advisor in Rare Diseases & Special Advisor, Openflex Ltd

×

Sheela Upadhyaya / Keynote: How do we reinvent the healthcare system to meet the demand of not only rare diseases, but potentially all patients in future?

Sheela Upadhyaya, Life Science Advisor in Rare Diseases & Special Advisor,
Openflex Ltd

Sheela Upadhyaya is a Life Sciences Consulant and a renowed expert in rare disease with over 25 year experience in the healthcare industry.  She has broad international experience spanning, UK including Scotland and the EU5 with a record of delivering leading edge innovation for rare diseases. ...

CloseMore

Monica Ines, HEOR Team Leader, Hemophilia, Pfizer

×

Monica Ines / Keynote: How do we reinvent the healthcare system to meet the demand of not only rare diseases, but potentially all patients in future?

Monica Ines, HEOR Team Leader, Hemophilia,
Pfizer

Mónica Inês is currently the HTA Value & Evidence Team Lead for GTx Hemophilia at Pfizer. Mónica has over 25 years of experience in the pharmaceutical industry, HEOR consulting companies and health care national payers, across several therapeutic areas and geographies. She is a health economist ...

CloseMore

Rachel Yang, Senior Advisor, Zhongshan Hospital Fudan University

×

Rachel Yang / Keynote: How do we reinvent the healthcare system to meet the demand of not only rare diseases, but potentially all patients in future?

Rachel Yang, Senior Advisor,
Zhongshan Hospital Fudan University

Rachel Yang, MD, PhD, a senior advisor at Fudan University, Zhongshan Hospital Rare Disease Center. She’s also served an advisory role at China Alliance for Rare Diseases (CHARD), and Chinese Organization for Rare Disorders (CORD). Rachel entered rare diseases serendipitously in 2015 and was...

CloseMore

Antoine Daher, President, Casa Hunter