August holds significant importance for our community as we unite to advance spinal muscular atrophy (SMA) care and research, and to raise awareness of the disease. Observing SMA Awareness Month throughout August, we aim to raise awareness, educate, advocate, and support those affected by SMA. This annual campaign serves as a beacon of hope, fostering awareness and driving fundraising efforts to find a cure while empowering those living with SMA to live life to the fullest.
Why Spinal Muscular Atrophy Awareness Month is Important
- Raising Awareness
Awareness campaigns like this can benefit people living with SMA by educating those unfamiliar with the condition and providing valuable information to those unsure of where to seek help, guiding them towards appropriate care and support.
- Showing Support
SMA can place a considerable emotional and financial strain on individuals and families living with the disease. Easing this burden can enhance their quality of life. Awareness campaigns play a crucial role in this, by fostering community support, driving policy changes, and securing funding for vital resources.
- Improving Treatments and Care
There is no cure for SMA. However, significant strides have been made in managing and treating the condition. The development of therapies like Spinraza® (Nusinersen), Evrysdi® (Risdiplam) and Zolgensma® (onasemnogene abeparvovec-xioi) shows the potential of research and scientists are working towards finding a cure. Increased attention and funding for research will bring us closer to that goal.
Supporting the Advancement of SMA Diagnosis, Care, and Treatment
At TREAT-NMD, our vision is to accelerate the development of effective treatments and establish best practice diagnosis and care for neuromuscular patients worldwide. We are internationally recognised for our role in addressing major issues in therapeutic development for neuromuscular diseases, including SMA.
Our contributions to the SMA community:
We offer a wealth of resources for patients, clinicians, and industry stakeholders. Our education projects for clinicians include masterclasses and e-learning modules, providing up-to-date information on diseases, patient registries, and the latest developments in neuromuscular research.
The TREAT-NMD Global Registry Network brings together independent neuromuscular disease (NMD) patient registries worldwide. This federated network currently includes 37 registries that collect data about more than 8,500 SMA patients around the world. In 2022 the registry network received a Letter of Support from the European Medicines Agency’s CHMP (Committee for Medicinal Products for Human Use) and this is guiding us as we develop plans to ask for Qualification of the registry network from the EMA.
By providing essential data, the TREAT-NMD Global Registry Network supports key developments in SMA research. The network recently completed a natural history study in spinal muscular atrophy (SMA) for the European Medicines Agency (EMA) and is working on other important SMA projects to monitor drugs in the real-world outside clinical trials.
The TREAT-NMD Advisory Committee for Therapeutics (TACT) plays an important role in the development of SMA therapies by offering a forum for expert, independent advice. Comprising of leading international experts, TACT evaluates drug development programs and offers comprehensive assessments and strategic guidance. This input helps streamline the drug development process, ultimately accelerating the availability of new therapies to patients.
The SMA clinical care guides offer vital information for patients, families, and healthcare professionals. TREAT-NMD translated these guides into 10 languages to ensure comprehensive care for SMA patients. Updated standards of care, reflecting new evidence and advancements, continue to improve the quality of life and life expectancy for those affected by SMA.
We support efforts to develop standardised operating procedures (SOPs) to unify experimental protocols, improving the comparability of studies across different laboratories. This standardisation is crucial for advancing research and developing new treatments.
Join Us
During SMA Awareness Month, we encourage everyone to get involved. You can participate by advocating, fundraising, educating, and raising awareness about SMA. Your support helps drive research and improve patient care.
Together, we can make a difference. Join TREAT-NMD in supporting SMA Awareness Month 2024, and help us bring greater attention to the needs, hopes, and experiences of the SMA community, while honouring the diverse individuals and perspectives of those living with the condition.
