24 scientific abstracts reflect UCB's ongoing commitment to improving outcomes for people living with neurological conditions, including rare epilepsies Dravet syndrome and Lennox-Gastaut syndrome, as well as generalized myasthenia gravis and thymidine kinase 2 deficiency.
Research includes disease course data of untreated patients with thymidine kinase 2 deficiency from largest international dataset.
Also features new data on generalized myasthenia gravis treatments RYSTIGGO®1 (rozanolixizumab-noli) and ZILBRYSQ®2 (zilucoplan) including an open-label extension study investigating rozanolixizumab self-administration and a phase 3 study on the effect of zilucoplan on specific outcome scores.
Data on FINTEPLA®3 (fenfluramine), BRIVIACT®4 (brivaracetam) CV, and investigational therapy STACCATO®* alprazolam showcase commitment to people living with epilepsies and their unmet needs.
BRUSSELS, April 3, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced it will present 24 abstracts from its expansive neurology portfolio, regarding rare epilepsies Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), generalized myasthenia gravis (gMG), and thymidine kinase 2 deficiency (TK2d), at this year's American Academy of Neurology (AAN) meeting, San Diego, California, April 5-9, 2025.
"We at UCB are deeply committed to driving innovation to improve the treatment and care of people living with severe neurological and neuromuscular conditions such as epilepsy, gMG, and TK2d. The data presented at AAN is a testament to our dedication to exploring new frontiers of science and improving care in areas of high unmet need. It is our mission to address critical gaps in care and bring meaningful solutions that make real improvements in the lives of the people we serve, now and into the future," said Donatello Crocetta, Chief Medical Officer at UCB.
Highlights of data to be presented at AAN include:
Epilepsy
DS and LGS: the final long-term safety and effectiveness data from a fenfluramine (FFA) open-label extension (OLE) study of 247 participants with LGS6, data from an observational analysis assessing sleep apnea association with increased mortality in patients 1-17 years old with severe epilepsy7, and data from a Phase 1 study to assess the safety, tolerability, pharmacokinetics, and efficacy of fenfluramine when combined with cannabidiol in a small cohort of patients with DS or LGS.8
Focal onset seizures: data include brivaracetam long-term clinical outcomes in pediatric patients with primary generalized seizures* from an open-label Phase 3 follow-up study9 and healthcare resource utilization of brivaracetam monotherapy† from a claims analysis.10
Women of childbearing age: data include results of a social media listening analysis on the experiences and challenges of women living with epilepsy during their motherhood journey.11
Quality of life: including data from a survey evaluating the disruptive impact of developmental and epileptic encephalopathies on patients' and families' quality of life12 and data from a study exploring the impact of prolonged seizures on patients' and caregivers' quality of life.13
UCB pipeline: data include investigational therapy STACCATO alprazolam*, being studied in management of stereotypical prolonged seizures.5,14
gMG
Rozanolixizumab self-administration study: Phase 3, open-label, randomized study which observed the safety and efficacy of manual push (MP) and syringe driver (SD) self-administration of rozanolixizumab was consistent with known profile.15
Ocular symptoms study in gMG: results from post hoc analyses from the randomized, placebo-controlled, Phase 3 MycarinG study investigating the effect of rozanolixizumab on ocular symptoms in patients with gMG.16
Study investigating switching to zilucoplan: Phase 3b study on safety, efficacy, and patient preference and satisfaction for subcutaneous zilucoplan in myasthenia gravis after switching from intravenous complement component 5 inhibitors.17
MG-specific subdomain scores: results from analysis from the randomized, placebo-controlled, Phase 3 RAISE study investigating the effect of zilucoplan on MG-ADL and QMG subdomain scores.18
Quality of life with gMG: a new international patient registry in gMG linking clinical and patient-reported outcomes data aims to improve understanding of the symptoms and quality of life to optimize future disease management.19
TK2d
Disease course: findings from the largest international TK2d dataset evaluating the disease course of people living with TK2d who were ≤12 years at TK2d symptom onset20 and over 12 at TK2d symptom onset and not on treatment.21
* STACCATO® alprazolam is an investigational treatment, and its safety and efficacy has not been established. It is not currently approved for use by any regulatory authority worldwide.
Rare Disease Connect in Neurology (RDCN)
UCB is proud to host its inaugural US Rare Disease Connect in Neurology (RDCN) Annual Summit at AAN, to provide a forum for needs-driven medical education for the gMG community. This event is being held exclusively for healthcare providers who are involved in patient care.
Symposia
AAN 2025 will feature two UCB-supported symposia:
Earlier identification and treatment of patients with a developmental and epileptic encephalopathy: taking a close look at Lennox-Gastaut syndrome: Saturday, April 5, 11:45 am-12:45 pm.
Rethinking seizure emergencies: expert perspectives on a new paradigm: Monday, April 7, 6 pm.
For further information, contact UCB:
Global Communications
Anna Clark
T: +44.73.8.668.67.79
email: [email protected]
Corporate Communications, Media Relations
Laurent Schots
T: +32.2.559.92.64
email: [email protected]
Rare Disease Communications
Daphne Teo
T: +1 (770) 880-7655
email: [email protected]
Epilepsy and Rare Syndromes Communications
Becky Malone
T: +1 (919) 605-9600
email: [email protected]
Investor Relations
Antje Witte
T: +32.2.559.94.14
email: [email protected]
About UCB
UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9 000 people in approximately 40 countries, the company generated revenue of € 6.15 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.
Forward-looking statements
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Important Safety Information about BRIVIACT (brivaracetam) in the US4
Indication
BRIVIACT® (brivaracetam) CV is indicated for the treatment of partial-onset seizures in patients 1 month of age and older.4
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Suicidal Behavior and Ideation: Antiepileptic drugs, including BRIVIACT, increase the risk of suicidal behavior and ideation. Monitor patients taking BRIVIACT for the emergence or worsening of depression; unusual changes in mood or behavior; or suicidal thoughts, behavior, or self-harm. Advise patients, their caregivers, and/or families to be alert for these behavioral changes and report them immediately to a healthcare provider.
Neurological Adverse Reactions: BRIVIACT causes somnolence, fatigue, dizziness, and disturbance in coordination. Monitor patients for these signs and symptoms and advise them not to drive or operate machinery until they have gained sufficient experience on BRIVIACT.
Psychiatric Adverse Reactions: BRIVIACT causes psychiatric adverse reactions, including non-psychotic and psychotic symptoms in adult and pediatric patients. Advise patients to report these symptoms immediately to a healthcare provider.
Hypersensitivity: BRIVIACT can cause hypersensitivity reactions. Bronchospasm and angioedema have been reported. Discontinue BRIVIACT if a patient develops a hypersensitivity reaction after treatment. BRIVIACT is contraindicated in patients with a prior hypersensitivity reaction to brivaracetam or any of the inactive ingredients.
Withdrawal of Antiepileptic Drugs: As with all antiepileptic drugs, BRIVIACT should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus.
DOSING CONSIDERATIONS
Dose adjustments are recommended for patients with all stages of hepatic impairment.
When BRIVIACT is co-administered with rifampin, an increase in the BRIVIACT dose is recommended.
ADVERSE REACTIONS
In adult adjunctive therapy placebo-controlled clinical trials, the most common adverse reactions (at least 5% for BRIVIACT and at least 2% more frequently than placebo) were somnolence and sedation, dizziness, fatigue, and nausea and vomiting symptoms. Adverse reactions reported in clinical studies of pediatric patients were generally similar to those in adult patients. Adverse reactions with BRIVIACT injection in adult and pediatric patients were generally similar to those observed with BRIVIACT tablets. Other adverse events that occurred in adult patients who received BRIVIACT injection included dysgeusia, euphoric mood, feeling drunk, and infusion site pain.
BRIVIACT is a Schedule V controlled substance.
Please refer to the full Prescribing Information.
Important Safety Information about FINTEPLA (fenfluramine) in the US3
INDICATIONS
FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.3
IMPORTANT SAFETY INFORMATION
BOXED WARNING: VALVULAR HEART DISEASE and PULMONARY ARTERIAL HYPERTENSION
There is an association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease and pulmonary arterial hypertension.
Echocardiogram assessments are required before, during, and after treatment with FINTEPLA.
FINTEPLA is available only through a restricted program called the FINTEPLA REMS.
CONTRAINDICATIONS
FINTEPLA is contraindicated in patients with hypersensitivity to fenfluramine or any of the excipients in FINTEPLA and with concomitant use, or within 14 days of the administration, of monoamine oxidase inhibitors because of an increased risk of serotonin syndrome.
WARNINGS AND PRECAUTIONS
Valvular Heart Disease and Pulmonary Arterial Hypertension (see Boxed Warning): Because of the association between serotonergic drugs with 5–HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), cardiac monitoring via echocardiogram is required prior to starting treatment, during treatment, and after treatment with FINTEPLA concludes. Cardiac monitoring via echocardiogram can aid in early detection of these conditions. In clinical trials for DS and LGS of up to 3 years in duration, no patient receiving FINTEPLA developed VHD or PAH.
Monitoring: Prior to starting treatment, patients must undergo an echocardiogram to evaluate for VHD and PAH. Echocardiograms should be repeated every 6 months, and once at 3-6 months post treatment with FINTEPLA.
The prescriber must consider the benefits versus the risks of initiating or continuing treatment with FINTEPLA if any of the following signs are observed via echocardiogram: valvular abnormality or new abnormality; VHD indicated by mild or greater aortic regurgitation or moderate or greater mitral regurgitation, with additional characteristics of VHD (eg, valve thickening or restrictive valve motion); PAH indicated by elevated right heart/pulmonary artery pressure (PASP >35 mmHg).
FINTEPLA REMS Program (see Boxed Warning): FINTEPLA is available only through a restricted distribution program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified by enrolling in the FINTEPLA REMS. Prescribers must counsel patients receiving FINTEPLA about the risk of VHD and PAH, how to recognize signs and symptoms of VHD and PAH, the need for baseline (pretreatment) and periodic cardiac monitoring via echocardiogram during FINTEPLA treatment, and cardiac monitoring after FINTEPLA treatment. Patients must enroll in the FINTEPLA REMS and comply with ongoing monitoring requirements. The pharmacy must be certified by enrolling in the FINTEPLA REMS and must only dispense to patients who are authorized to receive FINTEPLA. Wholesalers and distributors must only distribute to certified pharmacies. Further information is available at or by telephone at 1-877-964-3649.
Decreased Appetite and Decreased Weight: FINTEPLA can cause decreases in appetite and weight. Decreases in weight appear to be dose related. Approximately half of the patients with LGS and most patients with DS resumed the expected measured increases in weight during the open-label extension studies. Weight should be monitored regularly during treatment with FINTEPLA, and dose modifications should be considered if a decrease in weight is observed.
Somnolence, Sedation, and Lethargy: FINTEPLA can cause somnolence, sedation, and lethargy. Other central nervous system (CNS) depressants, including alcohol, could potentiate these effects of FINTEPLA. Prescribers should monitor patients for somnolence and sedation and should advise patients not to drive or operate machinery until they have gained sufficient experience on FINTEPLA to gauge whether it adversely affects their ability to drive or operate machinery.
Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including FINTEPLA, increase the risk of suicidal thoughts or behaviors in patients taking these drugs for any indication. Patients treated with an AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behaviors, or any unusual changes in mood or behavior.
Anyone considering prescribing FINTEPLA or any other AED must balance the risk of suicidal thoughts or behaviors with the risks of untreated illness. Epilepsy and many other illnesses for which AEDs are prescribed are themselves associated with morbidity and mortality and an increased risk of suicidal thoughts and behaviors. Should suicidal thoughts and behaviors emerge during treatment, consider whether the emergence of these symptoms in any given patient may be related to the illness being treated.
Withdrawal of Antiepileptic Drugs: As with most AEDs, FINTEPLA should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse reaction, rapid discontinuation can be considered.
Serotonin Syndrome: Serotonin syndrome, a potentially life-threatening condition, may occur with FINTEPLA, particularly during concomitant administration of FINTEPLA with other serotonergic drugs, including, but not limited to, selective serotonin-norepinephrine reuptake inhibitors (SNRIs), selective serotonin reuptake inhibitors (SSRIs), tricyclic antidepressants (TCAs), bupropion, triptans, dietary supplements (eg, St. John's Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors [MAOIs], which are contraindicated with FINTEPLA), dextromethorphan, lithium, tramadol, and antipsychotics with serotonergic agonist activity. Patients should be monitored for the emergence of signs and symptoms of serotonin syndrome, which include mental status changes (eg, agitation, hallucinations, coma), autonomic instability (eg, tachycardia, labile blood pressure, hyperthermia), neuromuscular signs (eg, hyperreflexia, incoordination), and/or gastrointestinal symptoms (eg, nausea, vomiting, diarrhea). If serotonin syndrome is suspected, treatment with FINTEPLA should be stopped immediately and symptomatic treatment should be started.
Increase in Blood Pressure
: FINTEPLA can cause an increase in blood pressure. Rare cases of significant elevation in blood pressure, including hypertensive crisis, has been reported in adult patients treated with fenfluramine, including patients without a history of hypertension. In clinical trials for DS and LGS of up to 3 years in duration, no pediatric or adult patient receiving FINTEPLA developed hypertensive crisis. Monitor blood pressure in patients treated with FINTEPLA.
Glaucoma: Fenfluramine can cause mydriasis and can precipitate angle closure glaucoma. Consider discontinuing treatment with FINTEPLA in patients with acute decreases in visual acuity or ocular pain.
ADVERSE REACTIONS
The most common adverse reactions observed in DS studies (incidence at least 10% and greater than placebo) were decreased appetite; somnolence, sedation, lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue, malaise, asthenia; ataxia, balance disorder, gait disturbance; blood pressure increased; drooling, salivary hypersecretion; pyrexia; upper respiratory tract infection; vomiting; decreased weight; fall; status epilepticus.
The most common adverse reactions observed in the LGS study (incidence at least 10% and greater than placebo) were diarrhea; decreased appetite; fatigue; somnolence; vomiting.
DRUG INTERACTIONS
Strong CYP1A2, CYP2B6, or CYP3A Inducers: Coadministration with strong CYP1A2, CYP2B6, or CYP3A inducers will decrease fenfluramine plasma concentrations. If coadministration of a strong CYP1A2, CYP2B6, or CYP3A inducer with FINTEPLA is necessary, monitor the patient for reduced efficacy and consider increasing the dosage of FINTEPLA as needed. If a strong CYP1A2, CYP2B6, or CYP3A inducer is discontinued during maintenance treatment with FINTEPLA, consider gradual reduction in the FINTEPLA dosage to the dose administered prior to initiating the inducer.
Strong CYP1A2 or CYP2D6 Inhibitors: Coadministration with strong CYP1A2 or CYP2D6 inhibitors will increase fenfluramine plasma concentrations. If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg. If a strong CYP1A2 or CYP2D6 inhibitor is discontinued during maintenance treatment with FINTEPLA, consider gradual increase in the FINTEPLA dosage to the dose recommended without CYP1A2 or CYP2D6 inhibitors. If FINTEPLA is coadministered with stiripentol and a strong CYP1A2 or CYP2D6 inhibitor, the maximum daily dosage of FINTEPLA is 17 mg.
USE IN SPECIFIC POPULATIONS
In patients with severe impairment of kidney function (estimated glomerular filtration rate [eGFR]) 15 to 29 mL/min/1.73m2, dosage adjustments are recommended. FINTEPLA has not been studied in patients with kidney failure (eGFR <15 mL/min/1.73m2).
Combined molar exposures of fenfluramine and norfenfluramine were increased in subjects with various degrees of hepatic impairment (Child-Pugh Class A, B, and C), necessitating a dosage adjustment in these patients.
To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 1–844-599-2273 or FDA at 1-800-FDA-1088 or .
Please refer to the full
Prescribing Information
.
Important Safety Information about
RYSTIGGO
(rozanolixizumab-noli)
in the US1
INDICATION
RYSTIGGO (rozanolixizumab-noli) is a neonatal Fc receptor blocker indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.1
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO administration in patients with an active infection until the infection is resolved. During treatment with RYSTIGGO, monitor for clinical signs and symptoms of infection. If serious infection occurs, administer appropriate treatment and consider withholding RYSTIGGO until the infection has resolved.
Immunization
Immunization with vaccines during RYSTIGGO treatment has not been studied. The safety of immunization with live or live-attenuated vaccines and the response to immunization with any vaccine are unknown. Because RYSTIGGO causes a reduction in IgG levels, vaccination with live-attenuated or live vaccines is not recommended during treatment with RYSTIGGO. Evaluate the need to administer age-appropriate vaccines according to immunization guidelines before initiation of a new treatment cycle with RYSTIGGO.
Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also called drug-induced aseptic meningitis) have been reported in patients treated with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.
Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and rash, were observed in patients treated with RYSTIGGO. Management of hypersensitivity reactions depends on the type and severity of the reaction. Monitor patients during treatment with RYSTIGGO and for 15 minutes after for clinical signs and symptoms of hypersensitivity reactions. If a reaction occurs, institute appropriate measures if needed.
ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of RYSTIGGO-treated patients) were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections were reported in 4% of patients treated with RYSTIGGO. Three fatal cases of pneumonia were identified, caused by COVID-19 infection in two patients and an unknown pathogen in one patient. Six cases of infections led to discontinuation of RYSTIGGO.
Please refer to the full Prescribing Information.
Important Safety Information about
ZILBRYSQ
(zilucoplan)
in the US2
INDICATION
ZILBRYSQ is a complement inhibitor indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.2
IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS
ZILBRYSQ, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis.
Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early.
Complete or update meningococcal vaccination (for serogroups A, C, W, and Y, and B) at least 2 weeks prior to the first dose of ZILBRYSQ, unless the risk of delaying therapy outweigh the risks of developing a meningococcal infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccination against meningococcal bacteria in patients receiving a complement inhibitor.
Patients receiving ZILBRYSQ are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected.
Because of the risk of serious meningococcal infections, ZILBRYSQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ZILBRYSQ REMS.
CONTRAINDICATIONS
ZILBRYSQ is contraindicated for initiation in patients with unresolved Neisseria meningitidis infection.
WARNINGS AND PRECAUTIONS
Serious Meningococcal Infections
ZILBRYSQ, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of ZILBRYSQ treatment is contraindicated in patients with unresolved serious Neisseria meningitidis infection.
Complete or update meningococcal vaccination (for serogroups A, C, W, Y and B) at least 2 weeks prior to administration of the first dose of ZILBRYSQ, according to current ACIP recommendations for patients receiving a complement inhibitor.
If urgent ZILBRYSQ therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible.
Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Consider interruption of ZILBRYSQ in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated.
ZILBRYSQ REMS
Due to the risk of meningococcal infections, ZILBRYSQ is available only through a restricted program under a REMS called ZILBRYSQ REMS.
Under the ZILBRYSQ REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risk of serious meningococcal infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated with meningococcal vaccines. Additional information on the REMS requirements is available at or 1-877-414-8353.
Other Infections
Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported in patients treated with complement inhibitors. ZILBRYSQ blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Administer vaccinations for the prevention of Streptococcus pneumoniae infection according to ACIP recommendations. Patients receiving ZILBRYSQ are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.
Pancreatitis And Other Pancreatic Conditions
Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ. Patients should be informed of this risk before starting ZILBRYSQ. Obtain lipase and amylase levels at baseline before starting treatment with ZILBRYSQ. Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved.
ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of gMG patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea.
Please refer to the full
Prescribing Information
.
BRIVIACT®, FINTEPLA®, RYSTIGGO® and ZILBRYSQ® are registered trademarks of the UCB Group of Companies. STACCATO® is a registered trademark of Alexza Pharmaceuticals, Inc., and is used by UCB Pharma under license.
©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-BR-2500017
1. Rystiggo® US PI. . Accessed February 2025.
2. Zilbrysq® US PI. Accessed February 2025.
3. Fintepla® US PI. . Accessed February 2025.
4. Briviact® US PI *1a42elk*_ga*NjA2OTM3NDAwLjE2ODI1ODQwNzc.*_ga_TXC8S80N6W*MTY5OTAyNTU2NS4yNy4xLjE2OTkwMjU1NzYuNDkuMC4w Accessed February 2025.
5. UCB acquires Engage Therapeutics: Staccato[®] Alprazolam - A potential solution for acute on-demand seizure management for people living with epilepsy. . Accessed February 2025.
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7. Dedeurwaerdere, et al. Sleep Apnea is Associated with High Mortality Risk in Children with Severe Epilepsies: Observational Analysis from Large Scale US Claims Data.
8. Zhang Roper, et al. Safety, Tolerability, Pharmacokinetics, and Efficacy of Fenfluramine in Combination With Cannabidiol: Results From a Phase 1 Study.
9. Lagae, et al. Long-term Tolerability and Efficacy of Adjunctive Brivaracetam in Pediatric Patients With Primary Generalized Seizures: Subgroup Analysis of an Open-label Follow-up Trial.
10. Bessons, et al. Brivaracetam Monotherapy Patient Characteristics, Treatment Patterns, and Healthcare Resource Utilization Among Patients With Epilepsy: A Cohort Study Using US Claims Data.
11. Baker, et al. Experiences of Women of Childbearing Age with Epilepsy Throughout their Motherhood Journey: Results From a Social Media Listening Study.
12. Bailey, et al. Disruptive Impacts of Developmental and Epileptic Encephalopathies on Patient and Family Life: a Quality-of-Life Survey.
13. Kaye, et al. Impact of Prolonged Seizures on Patients' and Caregivers' Quality of Life.
14. Daniels, et al. Inhalation as an Efficient Delivery Route of Alprazolam for the Treatment of Acute Seizures: Randomized Study of Staccato® Alprazolam Compared to Oral Alprazolam.
15. Bril, et al. Self-Administration of Rozanolixizumab in Patients With Generalized Myasthenia Gravis: The MG0020 Study.
16. Mahuwala, et al. Effect of Rozanolixizumab on Ocular Symptoms in Patients With Generalized Myasthenia Gravis: A Post Hoc Item-Level Analysis of Myasthenia Gravis-Specific Outcomes in MycarinG.
17. Freimer, et al. Switching to Subcutaneous Zilucoplan From Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction From a Phase 3b Study.
18. Weiss, et al. Effect of Zilucoplan on Myasthenia Gravis–Specific Outcome Subdomain Scores in RAISE: A Phase 3 Study.
19. Amini, et al. A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-Reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry.
20. Hirano, et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged ≤12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset.
21. Cristina Dominguez Gonzalez, et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged >12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset.
22. Nabbout, et al. Impact of Fenfluramine on Convulsive Seizure Frequency in Dose-Capped Patients With Dravet Syndrome.
23. Ameen, et al. Understanding the Incidence, Prevalence, Characteristics, and Healthcare Resource Utilization for Patients With Dravet and Lennox-Gastaut Syndromes.
24. Lhatoo, et al. Real-world Use of Fenfluramine for Dravet Syndrome: a Retrospective Cohort Study Using a National Pharmacy Database.
25. Bass, et al. Interim Results of the US Fenfluramine Oral Solution Cardiovascular Safety Registry Study.
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27. Ameen, et al. A Retrospective Claims Study Evaluating Mortality in Patients with Lennox-Gastaut Syndrome or Dravet Syndrome in the United States.
28. Pascuzzi, et al. Correlation Between MG Symptoms PRO and Existing MG-Specific Outcome Scores in the Phase 3 MycarinG Study: Post Hoc Analysis.
29. Thompson, et al. Solutions to Address the Unmet Needs of the gMG Patient Journey in the US: A Multistakeholder Delphi Consensus Study (RWE0970).
SOURCE UCB
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