SMA Awareness Month: Our Commitment to a Resilient Community

Improving the lives of people with rare neurological and neuromuscular diseases has been the focus of my life’s work for nearly two decades. As a clinical neurologist, I have seen firsthand the burden that patients and their families carry each and every day. Their journey inspires me to follow the promise of science to bring forward the bold medical discoveries that have the potential to fill unmet treatment needs and transform lives.

As vice president of clinical development at Biohaven, I am currently focused on the treatment of Spinal Muscular Atrophy (SMA), a rare, genetic neuromuscular disease that robs children and adults of physical strength. SMA attacks and destroys motor nerve cells, taking away the ability to walk, eat, and even breathe. ?Onset typically occurs in childhood.?It impacts four out of every 100,000 people and is the number one genetic cause of death for infants.

August is SMA Awareness Month which shines a spotlight on the hopes and needs of children and adults living with SMA and their families. Like many scientific and medical professionals who are dedicated to the treatment of SMA, I am optimistic that the future can indeed be more hopeful. Part of my enthusiasm surrounds the progress made to-date in advancing disease-modifying therapeutics for this challenging disorder. These treatments have made a difference in diminishing the burden and increasing survival. I am proud and energized by how far science has come in treating SMA.

In spite of this progress, there still is much more to be done to improve the lives of people living with SMA. While disease-modifying therapies continue to have an impact, they do not target the muscles themselves. This has left a significant unmet need for treatments that address the residual weakness and functional impairments, such as difficulty walking and completing activities of daily life, that many living with SMA experience.

Targeting the muscle is a newer approach in SMA drug development and one I am excited to spearhead as the clinical lead for Biohaven’s SMA program. We recently started a Phase 3 clinical trial for taldefgrobep alfa, an investigational, novel muscle-targeted therapy, studied in combination with other approved treatments. Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth.

Our Phase 3 study is called RESILIENT, which could not be a more perfect way to describe children and adults living with SMA and the SMA community. It is a community where family members, caregivers, advocates, and health care providers band together and work tirelessly to manage the daily needs of those living with SMA, raise awareness of the burden of SMA, and support continued medical advancement. And of course, the real heroes are the children and adults living with SMA, who soldier on in hope that better days are ahead.

With Biohaven, the SMA community has found a partner that matches their enduring commitment to improve lives and always puts the needs of patients first. Our patient-centric values embody all aspects of our SMA program, from the inclusive study design which does not restrict or limit participation based on background therapy, ambulatory status, or classification of SMA; to assisting trial participants with mobility limitations by offering the ability to receive the study drug at home as well as in the clinic.

As we pursue our mission, we know that the SMA community is waiting. Their resiliency deserves our urgency. I am proud to be part of our dedicated and compassionate team at Biohaven that is committed to following science toward a more hopeful future for people with SMA.?