Novartis Gene Therapies

Learn more about the key strategies and insights shaping the landscape of gene therapy in “Gene Therapies: Unlocking the opportunity for patient access”, a comprehensive white paper authored by our Global Value and Access team. After the successful approval and commercialization of the first gene therapy treatments worldwide, we review the challenges and successes in ensuring patient access across different markets, including five practices instrumental in ensuring patient access for these innovative treatments. Read the full paper for an in-depth exploration of gene therapies and their potential to transform patient access worldwide: bit.ly/3Ub3Ete #Novartis #NovartisGeneTherapies #GeneTherapy

We’re so proud of our Patient Advocacy team, who took home the Patient Advocacy Award at #AdvancedTherapiesWeek2024 last week. Their work in establishing deep and collaborative relationships with advocacy partners and the rare disease community is unmatched. Congratulations to the team!

 We’re thrilled to share this monumental achievement!   With your support, we’ve achieved our goal of 100 percent newborn screening for spinal muscular atrophy (SMA) in all 50 states. Now, every life born nationwide can benefit from early diagnosis and prompt access to treatments and care.   We want to thank our partner, Cure SMA, and the entire SMA community for your years of tireless advocacy. Together, we have shown the potential to positively impact the trajectory of those living with SMA. 

Did you know that nearly 1 in 50 people are carriers of the gene mutation for spinal muscular atrophy (SMA)?   #SMA is a rare, devastating genetic disease and, when left untreated, a leading genetic cause of infant death. It affects muscle functions, which can present in an infant as poor breathing and difficulty feeding/swallowing and difficulty with basic movement.    For parents and caregivers, tracking movements, also known as motor (or developmental) milestones, in the first few months of life is vital in helping monitor growth and identifying SMA early for timely intervention.    Learn more about the signs of SMA here: bit.ly/3ByYqNS   #SpinalMuscularAtrophy

For families who have received a new #SpinalMuscularAtrophy (SMA) diagnosis, know that you have help. Our partner Cure SMA provides practical support programs for the SMA community and advocates for their needs. Together, we strive to create a community where every individual is heard and feels welcomed.   Learn more about Cure SMA’s work: www.curesma.org     Photo credit: Cure SMA

Spinal muscular atrophy (SMA) is not always clinically evident at birth. Sometimes, its signs and symptoms are not present until later in an infant’s life, but by then, disease progression may have already caused irreversible damage.    SMA causes irreversible motor neuron loss affecting muscle functions, including breathing, swallowing and basic movement. Many other conditions and disorders share common symptoms with SMA, so it is important to identify the key signs to help with diagnosis and prompt treatment.   Learn more about SMA: https://bit.ly/3KP7oe0   #SpinalMuscularAtrophy #Novartis #SignsOfSMA 

What are the genetics of spinal muscular atrophy (SMA)? This rare disease is caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions.   Because SMA is a leading genetic cause of infant death, it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible.   Learn more about SMA here: bit.ly/3KP7oe0

On #CarersRightsDay we celebrate those who selflessly commit themselves to the care of their loved ones.   For people living with #SMA, a rare, devastating genetic disease, caregivers report that the inability to participate in activities, hip, thigh or knee weakness and fatigue are the symptoms that most impact children with SMA.   Supporting a child with SMA can require around the clock proactive supportive care. Taking on the role of caregiver for a child with SMA can put a tremendous strain on an individual’s social and professional life.   Learn more about our commitment to patients and their caregivers: bit.ly/3wossBI

#WorldChildrensDay offers a time in the year where we can come together to advocate, promote, and celebrate children’s rights across the globe including their right to access the best healthcare possible.    #SMA can cause devastating effects if left untreated. Diagnosis through newborn screening (#NBS) can help detect the disease early to support timely treatment.   In the spirit of World Children’s Day, our mission at Novartis Gene Therapies is to continue to collaborate with stakeholders to prioritize NBS programs worldwide to help babies with SMA gain access to life changing therapies.   Learn more about our commitment to improving access for babies with SMA here: bit.ly/3rek7RT

In the journey of caring for children with spinal muscular atrophy (SMA), we sometimes forget to acknowledge the value of caregivers. These remarkable individuals dedicate their lives to providing love, support and sometimes round-the-clock care to those affected by this condition.   Their unwavering dedication and sacrifices remind us of the profound impact that love and care can have on those who need it most.   bit.ly/3ncLA3H   #SpinalMuscularAtrophy #Caregivers #NationalFamilyCaregiversMonth