Drugs / Nusinersen
Nusinersen
also known as
BIIB058 · ISIS 396443 · ISIS-396443 SODIUM · ISIS-SMNRx · NUSINERSEN SODIUM · Spinraza
Antisense oligonucleotide targets SMN2
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed arthrogryposis spinal muscular atrophy 3 milestones | |||||
| Filed | EU (EMA) | — | arthrogryposis spinal muscular atrophy | 2025-01-23 | “the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA)” biogen.com ↗ |
| Filed | EU (EMA) | SPINRAZA | arthrogryposis spinal muscular atrophy | 2016-10-28 | “the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA)” biogen.com ↗ |
| Filed | US (FDA) | SPINRAZA | arthrogryposis spinal muscular atrophy | 2016-09-26 | “Earlier in September 2016, Biogen completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen.” biogen.com ↗ “its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA)” biogen.com ↗ “Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen” biogen.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (3) | US (FDA) | SPINRAZA | — | 2018-05-14 – 2026-03-27 | fda.gov ↗ |
| Approved | EU (EMA) | Spinraza | — | 2017-05-30 | europa.eu ↗ |
| Approved | US (FDA) | SPINRAZA | — | 2016-12-23 | fda.gov ↗ |
Trials 20
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT04050852 | Jul 2019 → Apr 2021 | spinal muscular atrophy | NYU Langone Health | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 14 trials | ||||||
| Phase 1 | NCT06555419 | Jan 2025 → Sep 2027 expected | spinal muscular atrophy | Biogen | Recruiting | No outcome recorded |
| Phase 1 | NCT02052791 | Jan 2014 → Jan 2017 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1 | NCT01780246 | Jan 2013 → Feb 2014 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1 | NCT01494701 | Nov 2011 → Jan 2013 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01703988 | Oct 2012 → Jan 2015 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 23 trials · 1 met primary | ||||||
| Phase 2 | NCT02462759 | Aug 2015 → Sep 2018 | spinal muscular atrophy | Biogen | Terminated | No outcome recorded |
| Phase 2 | NCT02386553 | May 2015 → Dec 2024 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 2 | NCT01839656 | May 2013 → Aug 2017 | spinal muscular atrophy | Biogen | Completed | Met primary |
| Phase 37 trials · 3 met primary | ||||||
| Phase 3 | NCT07846696 | Sep 2026 → Dec 2028 expected | spinal muscular atrophy | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT05067790 | Jan 2022 → Sep 2027 expected | spinal muscular atrophy | Biogen | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04729907 | Apr 2021 → Jul 2026 overdue | spinal muscular atrophy | Biogen | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04089566 | Mar 2020 → Feb 2024 | spinal muscular atrophy | Biogen | Completed | Met primary |
| Phase 3 | NCT02594124 | Nov 2015 → Aug 2023 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 3 | NCT02292537 | Nov 2014 → Feb 2017 | spinal muscular atrophy | Biogen | Completed | Met primary |
| Phase 3 | NCT02193074 | Aug 2014 → Nov 2016 | spinal muscular atrophy | Biogen | Terminated | Met primary Stop: Enrollment |
| Phase 41 trial | ||||||
| Phase 4 | NCT04488133 | Jan 2021 → Oct 2025 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase not applicable2 trials | ||||||
| — | NCT07838779 | Sep 2026 → Dec 2028 expected | spinal muscular atrophy | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | Not yet recruiting | No outcome recorded |
| — | NCT04576494 | Jan 2022 → Jan 2023 | spinal muscular atrophy | Reims University Hospital | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT02865109 | — | spinal muscular atrophy, type 1 | Biogen | No longer available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT07047144 Background | Sep 2025 → Nov 2028 expected | spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 45
| Date | Issuer | Release |
|---|---|---|
| 2026-03-05 | Biogen | Results Biogen to Share New SMA Data at Muscular Dystrophy Association and SMA Europe Conferences biogen.com ↗
Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) |
| 2026-02-04 | Biogen | Results Nature Medicine Publishes Results from the Pivotal DEVOTE Study of High-Dose Regimen of Nusinersen in Spinal Muscular Atrophy biogen.com ↗
The results showed the safety and effectiveness of the high-dose regimen of nusinersen across a broad range of people living with SMA, irrespective of age, prior treatment experience, and baseline functional status. |
| 2026-01-12 | Biogen | Regulatory Biogen Receives European Commission Approval for High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy biogen.com ↗
The SPINRAZA European Union marketing authorization has been updated to include the high dose regimen. |
| 2025-11-17 | Biogen | Regulatory High Dose Regimen of Nusinersen Receives Positive CHMP Opinion for the Treatment of Spinal Muscular Atrophy biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending by consensus the approval of the high dose regimen of nusinersen for the treatment of 5q spinal muscular atrophy (SMA). |
| 2025-09-23 | Biogen | Regulatory Biogen Provides Regulatory Update on High Dose Regimen of Nusinersen biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the Company’s supplemental New Drug Application (sNDA) for the high dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA). |
| 2025-06-27 | Biogen | Results New Data for Nusinersen Underscore Biogen’s Commitment to Advancing Clinical Research to Improve Outcomes in SMA biogen.com ↗
Final results from the landmark NURTURE study highlight the profound impact of early treatment with 12 mg SPINRAZA in clinically presymptomatic SMA with 92% of children achieving the ability to walk independently |
| 2025-01-23 | Biogen | Regulatory FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA biogen.com ↗ |
| 2024-10-08 | Biogen | Results New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA biogen.com ↗
Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen |
| 2024-09-04 | Biogen | Results Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA biogen.com ↗
The study met its primary endpoint at six months, achieving a statistically significant improvement in motor function in infants who received the higher dose regimen as compared to a prespecified matched sham (untreated) control group from the ENDEAR study. |
| 2024-03-06 | Biogen | Results New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy biogen.com ↗
The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA. |
| 2023-06-30 | Biogen | Results New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy biogen.com ↗
Interim results from the RESPOND study show improved motor function in most participants treated with SPINRAZA after Zolgensma ® (onasemnogene abeparvovec) |
| 2022-06-15 | Biogen | Results New Data Presented at Cure SMA Reveal Residual Unmet Needs in Young SMA Patients Treated With Gene Therapy and Suggest Further Potential of Using SPINRAZA® (nusinersen) biogen.com ↗
Final safety data from DEVOTE Part A support the continued development of an investigational, higher dose of nusinersen; additional study phases are actively enrolling |
| 2022-03-14 | Biogen | Results Biogen Announces New Updates Across its SMA Research Program at 2022 MDA Conference biogen.com ↗
New NURTURE results continue to show the potential long-term benefit of early treatment before SMA symptom onset, with 92 percent of participants now able to walk alone, most in age-appropriate timelines |
| 2021-06-10 | Biogen | Results New Data at Cure SMA 2021 Highlight the Long-Term Efficacy of SPINRAZA® (nusinersen) and Biogen’s Commitment to Innovation in SMA Therapy biogen.com ↗
Among children and teens with later-onset SMA in the SHINE study, long-term treatment with SPINRAZA improved walking distance and reduced fatigue |
| 2021-04-19 | Biogen | Results Biogen Advances Leading Research in Spinal Muscular Atrophy (SMA) with New Data at AAN 2021 Exploring Opportunities to Improve Outcomes for Patients biogen.com ↗
Initial findings from the DEVOTE study suggest no new safety concerns and support continued development of a higher dose of SPINRAZA ® (nusinersen) |
| 2020-06-10 | Biogen | Results New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA® (nusinersen) Continue to Demonstrate Sustained Benefit From Treatment biogen.com ↗
In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. |
| 2020-05-18 | Biogen | Results New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of Spinal Muscular Atrophy (SMA) Patients biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the sustained efficacy and longer-term safety of SPINRAZA in a broad range of patients with spinal muscular atrophy (SMA). |
| 2019-10-02 | Biogen | Results Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with SPINRAZA® (nusinersen) Published in Neuromuscular Disorders biogen.com ↗
Data from the NURTURE study demonstrated that infants who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained unparalleled results compared to the natural history of the disease. |
| 2019-09-18 | Biogen | Results Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study Evaluating a Higher Dose of SPINRAZA® (nusinersen) and Additional Data in a Broad Range of Patients biogen.com ↗
Long-term data from the SHINE study demonstrate improvements in or stabilization of motor function in patients as old as 21 treated with SPINRAZA for up to nearly six years |
| 2019-07-01 | Biogen | Results Biogen Announces New Data Further Establishing SPINRAZA® (nusinersen) as a Foundation of Care in Spinal Muscular Atrophy for a Broad Range of Patients biogen.com ↗
These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease. |
| 2019-05-14 | Biogen | Regulatory Biogen’s SPINRAZA® (nusinersen) Receives Positive Recommendation from NICE for Funding in the United Kingdom for the Treatment of Infants, Children and Adults with Spinal Muscular Atrophy biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that The National Institute for Health and Care Excellence (NICE) in the United Kingdom has recommended funding for SPINRAZA (nusinersen) on the National Health Service (NHS). |
| 2019-05-06 | Biogen | Results Biogen Presents Data at 2019 AAN Annual Meeting Affirming Longer-Term Safety and Durability of Treatment with SPINRAZA® (nusinersen) biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced new data affirming the safety and durability of SPINRAZA ® (nusinersen) and highlighting its clinically meaningful benefits for individuals with spinal muscular atrophy (SMA). |
| 2019-05-01 | Biogen | Results Biogen To Present Data At AAN Highlighting Its Innovative Marketed Treatments And Investigational Pipeline Programs For Complex Neurodegenerative Diseases biogen.com ↗
Data demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA ® (nusinersen) over three years achieved motor milestones that are more consistent with normal childhood development |
| 2019-04-30 | Biogen | Results Data Published in Neurology Show that Treatment with SPINRAZA® (nusinersen) Improved Motor Function and Provided Long-Term Benefit for Individuals with Later-Onset Spinal Muscular Atrophy biogen.com ↗
The data show that individuals with later-onset SMA, treated with SPINRAZA, regained motor function that had been previously lost and that treatment stabilized their disease activity leading to improvements in activities of daily living. |
| 2019-04-12 | Biogen | Results Biogen Continues to Advance Innovation in SMA with New Data in Adults and Infants to Be Presented at MDA Clinical and Scientific Conference biogen.com ↗
Infants with pre-symptomatic SMA treated with SPINRAZA® (nusinersen) achieve motor milestones that are more consistent with normal development, with 100 percent sitting independently as reported in the NURTURE study |
| 2019-02-28 | Biogen | Regulatory Biogen Further Expands Presence in China with Approval of SPINRAZA® (nusinersen), the First and Only Treatment for Spinal Muscular Atrophy biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that SPINRAZA (nusinersen) has been approved by the China National Medical Products Association (NMPA) for the treatment of 5q spinal muscular atrophy (SMA), expanding the company’s presence in China. |
| 2018-10-06 | Biogen | Results New SPINRAZA® (nusinersen) Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with Spinal Muscular Atrophy biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA ® (nusinersen) in 25 presymptomatic infants with SMA. |
| 2018-03-12 | Biogen | Results New Data Presented at MDA Clinical Conference Show Benefit in Motor Function for Infants, Teens and Young Adults Treated With SPINRAZA® (nusinersen) biogen.com ↗
In NURTURE, all infants treated with SPINRAZA were alive, did not require permanent ventilation and showed improvement in motor function and motor milestone achievements as of July 5, 2017, compared to the disease’s natural history. |
| 2018-02-14 | Biogen | Results The New England Journal of Medicine Publishes SPINRAZA® (nusinersen) Phase 3 Study Results in Individuals with Later-Onset Spinal Muscular Atrophy biogen.com ↗
The full manuscript titled, “Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy,” appears in the February 15 issue of The New England Journal of Medicine . |
| 2017-11-01 | Biogen | Results The New England Journal of Medicine Publishes First Phase 3 Study Results of SPINRAZA® for the Treatment of Spinal Muscular Atrophy biogen.com ↗ |
| 2017-10-05 | Biogen | Results Biogen’s SPINRAZA® (nusinersen) Data Show Earlier Treatment Initiation May Lead to Improved Motor Function Across a Broad Population of People Living with Spinal Muscular Atrophy biogen.com ↗
A new analysis from the Phase 3 ENDEAR study showed infants with SMA who initiated treatment earlier in the disease (shorter disease duration) demonstrated greater benefit and improvement in motor function outcomes. |
| 2017-06-29 | Biogen | Results New Data Reaffirm Clinically Meaningful Benefit of SPINRAZA® (nusinersen) in Individuals with Spinal Muscular Atrophy Across Disease Severity biogen.com ↗
We are encouraged to see unprecedented motor function gains in infants on permanent ventilation and a continued favorable benefit-risk profile across a broad population including no increase in risk of adverse events in children who have developed scoliosis. |
| 2017-06-01 | Biogen | Regulatory SPINRAZA® (Nusinersen) Approved in the European Union as First Treatment for Spinal Muscular Atrophy biogen.com ↗
The European Commission (EC) has granted a marketing authorization for SPINRAZA ® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), Biogen (NASDAQ:BIIB) announced today. |
| 2017-04-24 | Biogen | Results Final Phase 3 Study Data Show SPINRAZA® (nusinersen) Significantly Improved Motor Function in Children with Later-Onset Spinal Muscular Atrophy biogen.com ↗
The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. |
| 2017-04-21 | Biogen | Regulatory Biogen’s SPINRAZA® (nusinersen) Receives Positive CHMP Opinion for the Treatment of Spinal Muscular Atrophy biogen.com ↗
Biogen (NASDAQ: BIIB) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the granting of a marketing authorization for SPINRAZA ® (nusinersen) to treat patients with spinal muscular atrophy (SMA). |
| 2017-04-18 | Biogen | Results Biogen Highlights Advances from Its Neurology Research Programs and Portfolio of Innovative Medicines at AAN Congress biogen.com ↗
New SPINRAZA ® Data Show Robust Efficacy and Safety Across a Broad Range of Individuals with SMA |
| 2017-01-13 | Biogen | Results New Data Show SPINRAZA™ (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation in Infantile-Onset Spinal Muscular Atrophy biogen.com ↗
Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA™ (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA) compared to untreated infants. |
| 2016-12-23 | Biogen | Regulatory U.S. FDA Approves Biogen’s SPINRAZA™ (nusinersen), The First Treatment for Spinal Muscular Atrophy biogen.com ↗
The U.S. Food and Drug Administration (FDA) approved Biogen’s (NASDAQ: BIIB) SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. |
| 2016-11-07 | Biogen | Results Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular Atrophy biogen.com ↗
The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. |
| 2016-10-28 | Biogen | Regulatory Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA biogen.com ↗
The regulatory review process for these applications has now been initiated in the U.S. and EU. |
| 2016-10-08 | Biogen | Results New Data Presented at World Muscle Society Congress Support Potential Benefit of Investigational Treatment Nusinersen in Spinal Muscular Atrophy biogen.com ↗
In this analysis, infants treated for up to one year achieved motor milestones in timelines more consistent with normal development than what is observed in the natural history of patients with Type 1 SMA. |
| 2016-09-26 | Biogen | Regulatory Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy biogen.com ↗
Biogen (NASDAQ: BIIB) and Ionis (NASDAQ:IONS) today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). |
| 2016-08-01 | Biogen | Results Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy biogen.com ↗
The analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. |
| 2014-02-21 | Ionis Pharmaceuticals | Results Isis Pharmaceuticals Reports Interim Results from ISIS-SMN Rx Phase 2 Study in Infants with Spinal Muscular Atrophy ionis.com ↗
All four infants are alive and none have required permanent respiratory assistance. |
| 2014-02-21 | Ionis Pharmaceuticals | Results Isis Pharmaceuticals Reports Interim Results from ISIS-SMN Rx Multiple Dose Study in Children with Spinal Muscular Atrophy ionis.com ↗
Consistent with single-dose observations, time- and dose-dependent increases in muscle function were observed in children treated with multiple doses of ISIS-SMNRx. |
All press releases naming this drug 65 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 24 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | nusinersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02594124 ↗ |
| Known as | BIIB058 | “An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With...” NCT07838779 ↗1“An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)” NCT07846696 ↗ |
| Known as | ISIS 396443 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | ISIS-396443 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | ISIS-SMNRx | “Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy.” PMID 26823478 ↗ Jan 2016 |
| Known as | NUSINERSEN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | Spinraza | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Action | Restore | “thus increase the amount of functional survival motor neuron (SMN) protein that is deficient in patients with spinal muscular atrophy” PMID 27939059 ↗ Dec 2016 |
| Modality | Antisense oligonucleotide | “is an antisense oligonucleotide drug” PMID 26823478 ↗ Jan 2016 |
| Route | Intrathecal | “administered intrathecally” NCT01494701 ↗ |
| Target | SMN2 | “specifically designed to alter splicing of SMN2 pre-mRNA” PMID 27939059 ↗ Dec 2016 |