Web15 okt. 2015 · Current development-stage programs include antisense drugs to treat patients with spinal muscular atrophy (SMA), ISIS-SMN Rx, myotonic dystrophy type 1 … WebIonis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients …
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Web12 apr. 2024 · Some of the treatment options for SMA include medications, such as nusinersen and onasemnogene abeparvovec, that aim to increase the production of the SMN protein. Physical therapy and assistive devices, such as braces and wheelchairs, may also manage symptoms and improve quality of life. Web9 jan. 2024 · SPINRAZA is the first approved therapy for the treatment of spinal muscular atrophy (SMA) and remains the global market leader and foundation of care for patients … small o rings for glasses
ISS-N1 makes the First FDA-approved Drug for Spinal Muscular …
WebSMA is a genetic disorder that starts in the central nervous system (CNS) and affects all the muscles in the body. Due to the degenerative nature of the disease, people with SMA will experience a decline in muscle strength over time, although the rate and severity can vary among individuals WebCompany. Ionis Pharmaceuticals, Inc. is engaged in discovering and developing ribonucleic acid (RNA)-targeted therapeutics. The Company is primarily focused on cardiovascular and neurology franchises. The Company’s products include SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA is for the treatment of patients with spinal … Web16 mrt. 2024 · Ionis Pharmaceuticals, Inc. houdt zich bezig met het ontdekken en ontwikkelen van ribonucleïnezuur (RNA)-gerichte therapeutica. De onderneming richt … small o2 tank back pack