The TREAT-NMD Advisory Committee for therapeutics (TACT) is a unique multi-disciplinary international group of academic and industry drug development experts as well as representatives of patient foundations and regulatory experts, who meet twice a year to review and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases.

Due to an increased demand for early advice, TACT is pleased to announce that it has now expanded its remit to be able to accept applications for advice from organisations or institutions in the very earliest stages of pre-clinical development. TACT has put together a pre-clinical panel of experts who can now advise on candidate selection and optimisation. We hope to pilot this new model within the next year, so if you think this could be of benefit to you and your programme please get in touch with Laura Robertson (laura.robertson@treat-nmd.com).

There are  two places per year for early advice and eight places per year to apply for advice after your lead candidate has been selected.  Currently, we still have two places available for this Autumn’s meeting which will take place in November this year and we will soon be accepting applications for our next in person meeting which will take place from the 21st to the 23rd of April in Philadelphia next year.

You can find out more information on TACT and how to apply via our website.

We are thrilled to welcome three new team members to the TREAT-NMD team this month.

Emma Watson and Seung Yeon Lee both joined the team as project managers. Before joining TREAT-NMD Emma worked for the UK Health Security Agency (UKHSA) as a Project Manager on a team working on the COVID-19 response.  Previously Seung worked as a business consultant at Sagentia Innovation, a technology and innovation consultancy, based in Cambridge, UK, and as an independent consultant while relocating to Newcastle from Cambridge.

In addition to Emma and Seung we are very happy to have welcomed Nathan Bent as our new Data Governance Manager. Before joining TREAT-NMD Nathan worked for believe housing, where he led Data Governance and Data Analytics. He was also responsible for Data Management including GDPR and related Privacy programmes, along with the implementation of a company-wide data strategy with, Data Innovation, Quality, Ethics and Privacy at its core. He is passionate about data in all its forms and how it can be used for good.

We have a fantastic line up of speakers for the upcoming TREAT-NMD Conference which will be held in Vancouver, Canada from the 7th to the 9th of December this year.  One of which is Nathan Peck who is the founder and CEO of Cure VCP Disease Inc. Check out the video below for a sneak preview of what Nathan will be talking about at the conference.

View the full programme or register via our TREAT-NMD conference website.

The TREAT-NMD Education Committee (TEC)  is very please to welcome its newest member Gustavo Dziewczapolski.

Established in 2018, the TREAT-NMD Education Committee (TEC) is a board of experts (academics and patient representatives) who are affiliated with TREAT-NMD and have an interest in the education events and materials that TREAT-NMD produces. This group has overall responsibility for overseeing the quality, independence and appropriateness of all educational materials and activities created by, linked to or endorsed by TREAT-NMD.

Dr. Dziewczapolski received his Master’s degree from the University of Buenos Aires, followed by a Ph.D. in Neuropharmacology. He then moved to the United States to hone his scientific career in neurosciences, spanning eighteen years in research at UC San Diego and the Salk Institute. Dr. Dziewczapolski joined Cure CMD, an Advocacy Patient Organization, as the new Scientific Director on September 1st 2016. He plays the role of liaison between researchers, clinicians, pharma industry, patients and families with the aim of accelerating CMD research, diagnostics, care standards, and translation of research discoveries into real therapies. His expertise and knowledge will be a great addition to the TEC.

The European Neuromuscular Centre has been organising workshops for over 25 years including themed workshops where special themes are identified each year prioritised by the needs of the neuromuscular patient and research community.

Workshops are delivered by experts in the field of neuromuscular conditions past topics include topics such as “Psychological Interventions for improving quality of life in slowly progressive neuromuscular disorders” and “Management of safety issues arising following AAV gene therapy”.

The next workshop – 2022 Genetic diagnosis, clinical classification, outcome measures, and biomarkers in Facioscapulohumeral Muscular Dystrophy (FSHD): relevance for clinical trials will take place face to face from September 30^th to October 2^nd .

Workshops are on invitational basis only. If you would like to attend an international ENMC workshop please mail the ENMC office at enmc@enmc.org with your motivational letter and a short CV. If a workshop has not yet reached its maximum number of participants, your request may be forwarded to the workshop organisers for consideration. This does not provide a guaranteed invitation to the workshop, but may lead to inclusion on a reserve list.

To find out more on upcoming workshops please visit the ENMC website

On September 23, 2022 at 10am EDT, a coalition of limb-girdle muscular dystrophy (LGMD) nonprofit organizations will lead an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD). This online event is open to the public with registration required. The meeting will be summarized in a Voice of the Patient report which, along with recorded footage of the meeting, will be publicly available.

This EL-PFDD will focus on six subtypes of LGMD: LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. The goal of this meeting is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to
FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

The meeting agenda will include speakers and panellists who are living with LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9, as well as caregivers of people living with these six subtypes. The morning sessions will focus on symptoms that matter most to them, and the toll these take on daily living. The afternoon session will include discussions on experiences with current treatments and attitudes towards future treatments, including tolerance to potential risks. For the first time, representatives from Coalition to Cure Calpain 3, CureLGMD2I, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation, have joined together to organize this event. Jointly referred to as the LGMD Coalition, these nonprofit organizations are encouraging members of the global patient community to make their voices heard. Individuals living with the subtypes of LGMD listed above, as well as their caregivers, are invited to participate in the meeting by partaking in live polling and by calling and writing in with comments. Academic investigators, clinicians, regulators, and industry representatives are encouraged to watch the meeting and incorporate patient/caregiver perspectives when developing and reviewing new therapies to best meet the needs and expectations of our community. Pre-registration is available here

A new issue of Journal of Neuromuscular Diseases (JND) has been published online. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases. Volume 9, Issue 4 has 10 articles, which includes an open access review article that compares deflazacort and prednisone/prednisolone in Duchenne muscular dystrophy, and a systematic review that provides a greater understanding of progression to loss of ambulation by limb-girdle muscular dystrophy subtype – along with eight research articles, one of which looks at long-term social and health impacts of the COVID-19 pandemic on people with muscular dystrophy (note: all IOS Press COVID-19 content is freely available via this page). Discover all the issue’s content here!

To be kept up to date with all of the latest JND news, you can sign up to the journal’s newsletter.

Issue link: https://content.iospress.com/journals/journal-of-neuromuscular-diseases/9/4   

This year the Myotonic Dystrophy Foundation (MDF) intends to issue up to two, two-year awards of up to $380,000 at $190,000 per award for early career scholars in the DM field for projects that focus on basic, translational and/or clinical research or care projects in myotonic dystrophy. The MDF recognizes the importance of recruiting and retaining basic science early career researchers, as well as the critical need to recruit and retain clinical early career researchers to focus on the field of DM.

Applications are due by September 9, 2022, with a start date for successful applicants of January 2023. Applicants are encouraged to contact MDF’s Chief Executive Officer, Dr. Tanya Stevenson, with any questions about this RFA or the scientific content of their proposals. Technical issues should be directed to MDF’s Research Coordinator Dr. Nadine Ann Skinner.

The 17th International Child Neurology Congress held in collaboration with the Turkish Child Neurology Association will take place from the 3rd to the 7th of October this year in Antalya, Turkey.

The congress will feature state of the art lectures delivered by world experts and cover topics such as the latest developments within different subspecialties of child neurology.

There will be plenty of interactive learning through breakfast seminars, workshops and masterclasses all aimed at clinicians and healthcare professionals.

New initiatives taking place at the congress include:

• “Controversies in Child Neurology” debates which will feature a seasoned moderator and two internationally recognized authorities debating the opposing sides of a controversy, including attendee participation
• A hands on workshop where small break out groups will have the chance to work on specific tasks together
• an ICNTN symposium for all members interested in contributing to international child neurology medical education and participating in the further development of the e-curriculum architecture and its evaluation tools.

The aim of the ICNA congress is to stimulate scientific activity, promote education and training, and to spread specialist knowledge within its community.

A full version of the programme and more information on how to register can be found on the ICNC website.

The Walk & Roll to Cure FSHD is an international event focused solely on funding progress for FSHD. Led entirely by dedicated volunteers, supported by staff, events take place all over the US and Canada.