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Muscular Disorders Drug Development Summit

Conference

What’s Happening?

Strengthening Translational and Clinical Development of a New Wave of Genetic, Stem Cell, Small Molecule and RNA-Based Muscle Disease Therapies
After a year marked by uncertainty around Sarepta's Elevidys, the field is on the verge of new era shaped by cross-modality innovation and meaningful progress, with FDA submissions anticipated from Regenxbio, Capricor and Avidity that could transform therapeutic options for muscular dystrophies.

With investments including Servier's $2.65 billion buyout of Edgewise signposting reignited pharma interest and funding opportunities, muscle disease drug developers are poised to double down on investments across their preclinical, translational and clinical programs in 2026 and beyond.

Take advantage of this timely opportunity to join 60+ industry experts spanning muscle disease biology, translation, clinical development and more at the Muscular Disorders Drug Development Summit for three days of focused collaboration designed to:

- Explore emerging modalities beyond gene therapy
- Discover lessons learnt from developers of approved drugs
- Enhance biomarker measurements of muscular function and activity
- Innovate more targeted delivery to specific muscles while avoiding off-target tissues
- Leverage real-world data to uncover patient-relevant endpoints

With momentum building, don't miss your chance to learn from Servier, Dyne Therapeutics, Sanofi, Biogen, Avidity Biosciences and more at the only forum dedicated to addressing biopharma's most pressing challenges in advancing novel therapies for muscular dystrophies, Pompe disease and related myopathies.

URLs:
Website: https://go.evvnt.com/3834160-0?pid=11150
Brochure: https://go.evvnt.com/3834160-3?pid=11150

Prices:
Full Access Pass: Conference + Workshop Day - Drug Developer Pricing: USD 3897.00,
Conference Only - Drug Developer Pricing: USD 2799.00,
Full Access Pass: Conference + Workshop Day - Academic Pricing: USD 3297.00,
Conference Only - Academic Pricing: USD 2399.00,
Full Access Pass: Conference + Workshop Day - Service Provider Pricing: USD 4897.00,
Conference Only - Service Provider Pricing: USD 3599.00

Speakers: Jun Shi, Director of Muscle Disease Research, Biogen, Ricardo Mondragon Gonzalez PhD, Principal Scientist, Muscle Biology and Rare Diseases, Sanofi, Kristina Haack, Senior Global Head Clinical Development Inherited Neurometabolic Diseases, Sanofi, Nitza Thomasson, Executive Director, Global Head of Research and Development, Servier, Jim Breitmeyer, Chief Executive Officer, Altay Therapeutics Inc., Domi Stickens, Chief Scientific Officer, Arrakis Therapeutics Inc., Barbora Malecova, Director of Biology, Avidity Biosciences, Inc., Jimmy Weterings, Vice President, Head Oligonucleotide Therapeutics, Bonito Biosciences Inc., Paige Martin, Associate Scientific Director, Critical Path Institute (C-Path), Thomas Natoli, Senior Director, Neuromuscular Research, Dyne Therapeutics, Natalie Hawryluk, Vice President, Drug Discovery and Early Development, Edgewise Therapeutics, Inc., Sweta Girgenrath, Vice President, Head of Cardiovascular and Neuromuscular and Cardiovascular Therapeutics, Entrada Therapeutics Inc., Mathias Schmidt, President and Chief Executive Officer, JCR Pharmaceuticals, Seemin Ahmed, Principal Scientist and Gene Therapy Leader, Novartis AG, Damon Wang, Chief Executive Officer, Nucyrna Therapeutics, Marla Weetall, Senior Vice President, Pharmacology and Biomarkers, PTC Therapeutics, Jiyeon Denninger, Medical Director, Clinical Development, Sarepta Therapeutics, Eva Chin, Executive Director, Solve FSHD

Strengthening Translational and Clinical Development of a New Wave of Genetic, Stem Cell, Small Molecule and RNA-Based Muscle Disease Therapies
After a year marked by uncertainty around Sarepta's Elevidys, the field is on the verge of new era shaped by cross-modality innovation and meaningful progress, with FDA submissions anticipated from Regenxbio, Capricor and Avidity that could transform therapeutic options for muscular dystrophies.

With investments including Servier's $2.65 billion buyout of Edgewise signposting reignited pharma interest and funding opportunities, muscle disease drug developers are poised to double down on investments across their preclinical, translational and clinical programs in 2026 and beyond.

Take advantage of this timely opportunity to join 60+ industry experts spanning muscle disease biology, translation, clinical development and more at the Muscular Disorders Drug Development Summit for three days of focused collaboration designed to:

- Explore emerging modalities beyond gene therapy
- Discover lessons learnt from developers of approved drugs
- Enhance biomarker measurements of muscular function and activity
- Innovate more targeted delivery to specific muscles while avoiding off-target tissues
- Leverage real-world data to uncover patient-relevant endpoints

With momentum building, don't miss your chance to learn from Servier, Dyne Therapeutics, Sanofi, Biogen, Avidity Biosciences and more at the only forum dedicated to addressing biopharma's most pressing challenges in advancing novel therapies for muscular dystrophies, Pompe disease and related myopathies.

URLs:
Website: https://go.evvnt.com/3834160-0?pid=11150
Brochure: https://go.evvnt.com/3834160-3?pid=11150

Prices:
Full Access Pass: Conference + Workshop Day - Drug Developer Pricing: USD 3897.00,
Conference Only - Drug Developer Pricing: USD 2799.00,
Full Access Pass: Conference + Workshop Day - Academic Pricing: USD 3297.00,
Conference Only - Academic Pricing: USD 2399.00,
Full Access Pass: Conference + Workshop Day - Service Provider Pricing: USD 4897.00,
Conference Only - Service Provider Pricing: USD 3599.00

Speakers: Jun Shi, Director of Muscle Disease Research, Biogen, Ricardo Mondragon Gonzalez PhD, Principal Scientist, Muscle Biology and Rare Diseases, Sanofi, Kristina Haack, Senior Global Head Clinical Development Inherited Neurometabolic Diseases, Sanofi, Nitza Thomasson, Executive Director, Global Head of Research and Development, Servier, Jim Breitmeyer, Chief Executive Officer, Altay Therapeutics Inc., Domi Stickens, Chief Scientific Officer, Arrakis Therapeutics Inc., Barbora Malecova, Director of Biology, Avidity Biosciences, Inc., Jimmy Weterings, Vice President, Head Oligonucleotide Therapeutics, Bonito Biosciences Inc., Paige Martin, Associate Scientific Director, Critical Path Institute (C-Path), Thomas Natoli, Senior Director, Neuromuscular Research, Dyne Therapeutics, Natalie Hawryluk, Vice President, Drug Discovery and Early Development, Edgewise Therapeutics, Inc., Sweta Girgenrath, Vice President, Head of Cardiovascular and Neuromuscular and Cardiovascular Therapeutics, Entrada Therapeutics Inc., Mathias Schmidt, President and Chief Executive Officer, JCR Pharmaceuticals, Seemin Ahmed, Principal Scientist and Gene Therapy Leader, Novartis AG, Damon Wang, Chief Executive Officer, Nucyrna Therapeutics, Marla Weetall, Senior Vice President, Pharmacology and Biomarkers, PTC Therapeutics, Jiyeon Denninger, Medical Director, Clinical Development, Sarepta Therapeutics, Eva Chin, Executive Director, Solve FSHD

When & Where
Jan 26, 2027, 9:00am to
Jan 28, 2027, 5:00pm Timezone: EST
$2,399.00


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