(Conference) Orphan Drugs and Rare Diseases – US Edition

Join us at our MarketsandMarkets Orphan Drugs and Rare Diseases Conference in Boston, USA for a groundbreaking exploration of the recent advances in developing life-saving therapies, technologies in diagnosing, and strategies to enhance orphan drug development.

The conference will focus on the wide scope of orphan drug developments, critical issues of elevated pricing, reimbursement & access to patients, and strategies to enhance access to novel diagnostics and effective therapies for rare to ultra–rare diseases.

Register Now @ https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/register

Conference Speakers-
1. Joslyn Arnon Crowe- Executive Director, National Niemann-Pick Disease Foundation
2. Donna (McKenna) Sullivan- Director of Patient Advocacy, Pathways To Trust
3. Zia Choudhry- Senior Medical Director Rare Autoantibody Diseases – Immunology, Johnson & Johnson
4. Adrian Kielhorn- Senior Director, Global HEOR Neurology, Alexion Pharmaceuticals, Inc
5. Karlie Sharma- Program Director, National Center for Advancing Translational Sciences, National Institutes of Health (NIH/NCATS)

……………. More

Become a speaker @ https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/speaker-enquiry

Key Sessions-
• Overview and Regulatory Updates: Current Landscape and Regulatory Developments in US Market.
• Emerging Therapies for RDs: Recent breakthroughs in CRISPR-Cas9 technology, Cell & Gene Therapies, mRNA therapies & Precision Treatments.
• Rare Disease and Genetics: Rare Genetic Disease Mechanisms and Therapeutic Targets.
• Patient Advocacy, Engagement & Collaboration: Critical Role of Patient Advocacy and Global Collaboration in Research and Development.
• Global Perspectives and Market Access: Global market dynamics and market access strategies, sustainable models & roadblocks for orphan drugs.
• ……more

Become a Sponsor @ https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/become-sponsor

About Us-
MarketsandMarkets Conferences, a leading international event organizer since 2014, takes pride in organizing over 200 conferences globally. With our extensive experience and rich legacy, we bring together experts from around the world to drive knowledge sharing and innovation in the healthcare and lifesciences sectors.

ADDRESS:
Magarpatta City, Hadapsar, Pune, India – 411013

1. MarketsandMarkets Orphan Drugs and Rare Diseases Conference – US Edition Date: 17th – 18 th October 2024 Address- Boston, USA For more details by calling [phone] or Send an email to [email protected] with your contact details and questions if any. 1 For more details [phone] or Send an email to [email protected] with your contact details and questions if any.

2. Orphan Drugs and Rare Diseases Conference Empowering Progress: Navigating Orphan Drugs & Rare Diseases from Regulation to Detection & Precision Treatments Join us at our MarketsandMarkets Orphan Drugs and Rare Diseases Conference in Boston, USA for a groundbreaking exploration of the recent advances in developing life-saving therapies, technologies in diagnosing, and strategies to enhance orphan drug development. Register Now: https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/register Speakers:- Joslyn Arnon Crowe Donna (McKenna) Sullivan Zia Choudhry Adrian Kielhorn …more 2

3. Orphan Drugs and Rare Diseases Conference The event will bring together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors, and solution providers. It is an unrivalled opportunity to network and exchange invaluable knowledge with the expert leaders in the rare disease community – patient advocates, policy makers, researchers, clinicians, healthcare professionals, healthcare industry representatives, academics, payers, regulators across the globe. Become a Speaker @ https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/speaker-enquiry Who Should Attend- Industry :  Pharmaceutical/Therapeutic and Biotechnology Companies Designations:  Chief Executives, President, Vice President, Director, Head of Department, Executive leader, Senior Manager, Manager, Principal Scientist, Scientists. Academic:  Hospitals, Universities, Research institutes – Professors & Researchers. ……MORE 3

4. Orphan Drugs and Rare Diseases Conference Key Highlights: Overview and Regulatory Updates : Current Landscape and Regulatory Developments in US Market. Emerging Therapies for RDs : Recent breakthroughs in CRISPR-Cas9 technology, Cell & Gene Therapies, mRNA therapies & Precision Treatments. Rare Disease and Genetics : Rare Genetic Disease Mechanisms and Therapeutic Targets. Patient Advocacy, Engagement & Collaboration : Critical Role of Patient Advocacy and Global Collaboration in Research and Development …… more Become a Sponsor @ https://events.marketsandmarkets.com/orphan-drugs-and-rare-diseases-conference-us-edition/become-sponsor 4

5. Orphan Drugs and Rare Diseases Conference For more details contact - [email protected] [phone] marketsandmarkets.com . MarketsandMarkets Conferences, a leading international event organizer since 2014, takes pride in organizing over 200 conferences globally. With our extensive experience and rich legacy, we bring together experts from around the world to drive knowledge sharing and innovation in the healthcare and lifesciences sectors. 5