SMA Type 4: Symptoms, Treatment, and More - SMA News Today
2023年2月2日 · One of the rarest forms of this rare disease, SMA type 4 accounts for less than 1% of all cases. It is also called adult-onset or late-onset SMA, because symptoms appear at a …
Oral SBMA therapy shows safety and tolerability in Phase 1 trial
2023年11月15日 · Results of the Phase 1 study (ACTRN12622000964718) were presented during the 2023 International Kennedy’s Disease Patient Conference held in London on Nov. …
Spinal muscular atrophy (SMA) symptoms – SMA News Today
2023年1月20日 · Because spinal muscular atrophy (SMA) is marked by the progressive loss of nerve cells needed for voluntary movement, symptoms such as muscle weakness, breathing …
Long-term Spinraza found safe, effective across SMA spectrum
2024年2月14日 · The data came from SMArtCARE, a registry that collects information from SMA patients in Germany, Switzerland, and Austria.It’s supported by the companies that market the …
FDA Approves Evrysdi, First Oral Treatment for All SMA Types
2020年8月7日 · Its list price, which is generally not the treatment’s cost to patients, is set at a maximum of $340,000 a year, according to Genentech.That price comes into effect for …
Spinraza, in Range of SMA Patients, Shows Efficacy Over Years of Use
2020年5月20日 · “As the first approved treatment for SMA, Spinraza offers a significant data set that allows us to uniquely assess the safety and durability of repeated doses over time …
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
2023年2月2日 · Type 2 is an intermediate form of spinal muscular atrophy (SMA), a rare genetic disease characterized by the progressive loss of motor neurons — the nerve cells controlling …
Real-world benefits of Zolgensma for SMA seen in RESTORE data
2024年1月31日 · RESTORE (NCT04174157) is a real-world, observational patient registry that’s tracking outcomes for up to 15 years and seeks to capture responses to DMTs that weren’t …
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
Spinal Muscular Atrophy (SMA) is an inherited neurodegenerative disease, 1 but it is increasingly clear that the pathology of SMA extends beyond ventral horn spinal cord neurons and includes …
SMA Type 0: Symptoms, Treatment, and More - SMA News Today
2023年2月2日 · What are the symptoms of SMA type 0? Spinal muscular atrophy type 0 is defined by symptoms that appear before birth, while a developing fetus is still in the womb. …