NMD Summit 2019: Advancing Drug Development for Neuromuscular Diseases

Running from October 23 to 25, in Boston, Massachusetts, USA, the Neuromuscular Drug Development (NMD) Summit 2019 will address translational challenges in the field of drug discovery and development for rare neuromuscular diseases.

With over 18 expert speakers, chances to network, share research, attend workshops and create new partnerships with peers, the NMD 2019 summit will focus on amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), among other rare diseases, to combat challenges in the industry.

There are a number of different events taking place across the whole summit, including expert speakers, workshops and a Mastermind session in which visitors can debate current industry issues. Attendees will also engage with research presentations from their peers to enhance learning, make connections and present new data and discoveries.

Cell body of a neuronSvitlana Pavliuk | Shutterstock

Why Attend the NMD Summit?

Established with Biogen, Audentes and PTC Therapeutics, the industry-focused summit will address and solve challenges faced in the development of neuromuscular drugs.

Visitors to the summit will be able to explore new research in, and approaches to, treating rare neuromuscular diseases, from gene therapy, antisense oligonucleotide (ASO) and small molecule treatments.

The clinical landscape itself will be readily analyzed and investigated to identify where improvements and advancements need to be made, and where regulation may hinder the realization of clinical expectations.

Along with this, visitors will have invaluable opportunities to learn from and share research, make connections with leaders in the field and gain insight into perspectives in neuromuscular disease research.

Join 50+ other neuromuscular stakeholders at this definitive forum to exchange novel ideas, gather lessons learned and establish new collaborations. Don’t miss your opportunity to be part of the force accelerating change in the wider therapeutic NMD landscape.


Workshop highlights include:

Effectively Utilizing Neuromuscular Biomarkers in Clinical Trials

23 October, 9AM – 12PM

Lead by Susan Ward, Head of Clinical Operations at Brainstorm Cell Therapeutics, this workshop will allow attendees to understand how biomarkers will interact with certain drugs and treatment, even delving into how common variables will affect the action of a treatment, from the time of day to the patient’s gender.

Linking the Regulatory and Reimbursement Landscape for Neuromuscular Disorder Drug Development

23 October, 12:30PM – 3:30PM

Bangor University Research Fellow Elin Haf Davies will be discussing what drug developers need to do in order to meet regulatory and reimbursement requirements. The FDA and EMA’s influence on the design of clinical trials will be explored, and attendees will be able to debate the cost of drugs, access, and reimbursement.

There will also be panel discussions including an assessment of the current approval considerations and regulations, investigating what changes need to be made in order to bring clinical expectations in line with current drug regulations. Speakers will also debate whether efficacy or biomarker improvement are more important in DMD and SMA approval.

Nerve cells showing chemical synapsesRost9 | Shutterstock

Expert Speaker Highlights

Below are just a handful of the fascinating range of expert speakers set to present at NMD Summit 2019. Topics range from patient advocacy and investment into rare diseases to challenges in drug development and delivery.

Accelerating Drug Development for Rare Diseases: Development of a Clinical Trial Simulation Tool for DMD

24 October, 3:30 PM

Jane Larkindale, Executive Director, Duchenne Regulatory Science Consortium of the Critical Path Institute, will speak on the challenges faced in enhancing the efficiency in developing drugs for rare diseases, with particular focus on DMD. Larkindale will present plans for the development of a clinical trial simulation tool for DMD, along with data and analysis. Similar tools to this will be discussed in order to determine how to develop tools for other rare diseases.

Is There Still a Role for Small Molecule Treatment for Neuromuscular Disorders?

25 October, 9:15AM

Kirsten Gruis, Global Head, Neuromuscular at Roche will be discussing if the role for small molecule treatments for neuromuscular disorders still remains. The talk will assess the advantages and disadvantages of these types of treatments and analyze two successes in utilizing small molecule treatments in rare diseases.

Patient Advocacy Partnerships to Improve Real World Evidence (RWE) in Neuromuscular Drug Development

25 October, 10:45AM

This talk, led by Shazia Ahmad of Patient and Physician Services, UBC, will review patient advocacy groups and the part they play in understanding rare diseases and developing drugs outside of clinical trials. The presentation will include panel members who are patient advocates themselves, representing neuromuscular diseases including DMD.

Networking Opportunities

Visitors to the NMD Summit will benefit from over seven hours of networking time to advance careers, make new connections, and form new partnerships.

There is a Speed Networking event during which visitors will be able to meet with key thought leaders in neuromuscular research, allowing them to benefit from new connections with the most active pharma and biotech organizations in the field.

Statistics for the types of companies attending the NMD Summit 2019 show that 30 percent of companies are large pharmaceutical companies, with 40 percent identifying at small biotech companies. Service providers and academic companies also make up a good portion of the attendees, guaranteeing visitors a wide range of industry contacts and perspectives.

The Poster Session

Thursday 24 October, 4:15PM

Muscle fiber - By Anton Nalivayko

The Poster Session will allow researchers to share work in their respective fields and establish their place in their area of research. It is a blend of networking and learning that allows visitors to present their research and discover new data in neuromuscular research and clinical development.

About the NMD Summit 2019

NMD summit 2019 LogoThe industry-focused 2nd Neuromuscular Drug Development Summit (NMD) will overcome the challenges preventing neuromuscular drug developers bringing truly transformative therapeutics to market.

With an emphasis on ALS, SMA, DMD and other rare diseases, 18 expert speakers from the likes of Biogen, Audentes and PTC Therapeutics will share their insights.

For more information, please contact [email protected]​hansonwade.​com or register for the event here.

Last updated: Sep 6, 2019 at 10:07 AM


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