Statements (58)
| Predicate | Object |
|---|---|
| gptkbp:instance_of |
gptkb:pharmaceuticals
|
| gptkbp:approves |
gptkb:FDA
December 2016 |
| gptkbp:availability |
prescription only
|
| gptkbp:brand |
gptkb:Spinraza
|
| gptkbp:capacity |
5 m L
|
| gptkbp:clinical_trial |
Phase 3
CHERISH ENDEAR NURTURE SHINE |
| gptkbp:clinical_use |
pediatric patients
adult patients |
| gptkbp:condition |
gptkb:spinal_muscular_atrophy_type_2
gptkb:spinal_muscular_atrophy_type_3 gptkb:spinal_muscular_atrophy_type_1 |
| gptkbp:contraindication |
coagulation disorders
active infection hypersensitivity to nusinersen |
| gptkbp:developed_by |
gptkb:Ionis_Pharmaceuticals
|
| gptkbp:dosage_form |
gptkb:Software_Solutions
|
| gptkbp:formulation |
sterile solution for injection
|
| https://www.w3.org/2000/01/rdf-schema#label |
Nusinersen
|
| gptkbp:impact |
enhances quality of life
improves motor function increases survival rates reduces need for respiratory support |
| gptkbp:invention |
patented
|
| gptkbp:loading_dose |
initial 4 doses
|
| gptkbp:marketed_as |
gptkb:Biogen
|
| gptkbp:mechanism_of_action |
antisense oligonucleotide
|
| gptkbp:patient_population |
gptkb:children
adults infants |
| gptkbp:pharmacokinetics |
half-life of approximately 4 hours
increases SMN protein levels |
| gptkbp:price |
high cost
|
| gptkbp:research |
ongoing studies for long-term effects
investigating combination therapies |
| gptkbp:research_areas |
neuromuscular disorders
|
| gptkbp:route_of_administration |
intrathecal injection
|
| gptkbp:service_frequency |
every 4 months after loading doses
|
| gptkbp:side_effect |
gptkb:pneumonia
headache nausea seizures constipation respiratory distress back pain injection site reactions thrombocytopenia elevated liver enzymes |
| gptkbp:storage |
refrigerated
|
| gptkbp:targets |
gptkb:SMN2_gene
|
| gptkbp:used_for |
treatment of spinal muscular atrophy
|
| gptkbp:website |
intrathecal space
|
| gptkbp:bfsParent |
gptkb:Biogen
|
| gptkbp:bfsLayer |
4
|