Jeff Guptill, MD, Neuromuscular Clinical Development Lead at Argenx, discusses the impact of the Vyvgart (efgartigimod alfa) approval on the myasthenia gravis (MG) community to treat patients with anti-MuSK-Ab positive, anti-LRP4-Ab positive, and/or triple seronegative MG.

MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles due to the presence of antibodies against the acetylcholine receptor. The exact reason this occurs is not known.

In May 2026, the US Food and Drug Administration (FDA) approved Vyvgart (efgartigimod alfa) and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) for the treatment of adult patients who have anti-MuSK-Ab positive, anti-LRP4-Ab positive, and/or triple seronegative MG. The medication was previously approved to treat patients with anti-AChR-Ab positive gMG.

Efgartigimod alfa is a first-in-class human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. Vyvgart Hytrulo is a subcutaneous combination of efgartigimod alfa and recombinant human hyaluronidase PH20 (rHuPH20) that allows for subcutaneous injection delivery.

Until this approval, traditional therapy was limited to cholinesterase inhibitors, corticosteroids and immunosuppressants for these MG subpopulations. However, research has shown that these subpopulations react to treatments in different ways, highlighting the need for more treatment options and research aimed at these specific subgroups.

Dr. Guptill explains the importance of this approval, specifically for the triple seronegative patients who now have their first rigorously studied treatment with a favorable safety and efficacy profile.

For more information on this approval visit, https://checkrare.com/fda-expands-indication-of-vyvgart-efgartigimod-alfa-for-adults-with-seronegative-myasthenia-gravis/

CHAPTERS
Introduction 00:00
Vyvgart (Efgartigimod Alfa) Indication 00:17
Antibody Presence’s Impact on Symptoms 00:47
Standard Therapy for Nontraditional Patients 1:52
Importance of Treatment Options 3:08
What Physicians Should Know 4:04

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Kerrie-Anne Ho, PhD, Patient Preference Lead at UCB, discusses preferences and perceptions of acute seizure medications.

Data from interviews with people with seizures (PwS) and caregiver interviews were presented at the American Academy of Neurology (AAN) 2026 Annual Meeting.

Patient and Caregiver Perceptions of Acute Seizure Medications and the Rapid and Early Seizure Termination (REST) Approach: Qualitative Interviews

The objective of this study was to assess understanding of the awareness of the Rapid and Early Seizure Termination (REST) paradigm and perceptions around acute (on-demand) medications among PwE and caregivers. REST is essential in preventing seizures from becoming prolonged seizures and progressing in severity.

A total of 53 participants (18 PwE, 35 caregivers) participated in 60-90-minute qualitative interviews regarding experiences with auras/epilepsy concepts/experience and unmet needs of acute medications/perceptions of REST. Of 44 participants who reported experiencing auras, 68% were extremely/very confident that they could predict a seizure from auras. The most frequent description of prolonged seizures given was longer than 5 minutes. Only 13% of participants did not use/administer benzodiazepines as acute medication for prolonged seizures. Regarding acute medication, 36% of participants desired to change the mode of administration and 23% wanted improved effectiveness.

53% of respondents perceived REST medication as quick/fast if it acts within the first minute of the seizure and 15% were aware of the REST concept. Of 45 participants not currently following the REST approach, 67% perceived REST to be feasible.

Patient and Caregiver Preferences for Acute Seizure Medications: A Quantitative Survey

The objective of this study was to explore the preferences of PwE and caregivers on attributes of acute (on-demand) seizure medications.

PwE aged 18 years and older who had experienced 1 or more prolonged seizures of 2 minutes or longer in the past 12 months and caregivers of PwE aged 12 years and older were included in the survey.

A total of 135 adult PwE and 239 caregivers participated (n=374). Overall, 46% of participants reported generalized or bilateral/tonic-clonic as their most common seizure type, 52% reported 3-5 seizures/month on average, and 57% reported an average duration of 1 to 4 minutes for the most common seizure type over the past 12 months. 57% of participants reported using oral acute medication for prolonged seizures.

The survey noted that all attributes significantly influenced treatment preferences, with the most important being time to seizure cessation and mode of administration. Nasal spray and single-use inhaler were most preferred, whereas rectal was least preferred. Participants preferred treating early in the seizure and faster-acting medications. Willingness-to-wait exercise showed that 88% of participants would be willing to wait 30 seconds (the minimum time proposed) to administer acute seizure medication.

CHAPTERS
Introduction 00:00
The REST Paradigm 00:25
Abstracts Presented at AAN 1:42
Theory on Waiting for Medication Administration 5:19
Next Steps 6:02

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Abigail Schwaede, MD, Assistant Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses results on intravenous efgartigimod in juvenile generalized myasthenia gravis (gMG).

gMG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. It mostly develops in adults but children can also develop this rare condition. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles due to the presence of antibodies against the acetylcholine receptor. The exact reason this occurs is not known.

The ADAPT JR (NCT04833894) clinical trial is a phase 2/3, open-label, uncontrolled study evaluating the pharmacokinetics, pharmacodynamics, safety, and activity of efgartigimod in children ages 2 to 18 years of age with acetylcholine receptor antibody–positive (AChR-Ab+) gMG. Efgartigimod is a human immunoglobulin G1 antibody Fc fragment, which blocks the neonatal Fc receptor. Phase 3 ADAPT/ADAPT+ trials demonstrated that efgartigimod IV is efficacious and well tolerated in adults with gMG.

During dose-confirmatory part A, participants received 1 efgartigimod infusion and pharmacokinetic, pharmacodynamic, and safety endpoints were monitored over 8 weeks. During treatment response–confirmatory part B, participants received 1 or 2 cycles of 4 once-weekly efgartigimod infusionsand additional endpoints, including MG Activities of Daily Living (MG-ADL) scores, were assessed over 18 weeks.

In the adolescent cohort, 6 participants enrolled in part A and continued to part B, and 5 participants enrolled directly in part B. Efgartigimod treatment resulted in IgG and AChR-Ab decreases similar to those observed in previous studies of adults with gMG. Safety profile and MG-ADL improvements were also similar to previous observations.

ADAPT JR child cohort enrollment is ongoing.

CHAPTERS
Introduction 00:00
Juvenile-Onset Myasythenia Gravis 00:14
Antibody Presence in Juvenile Disease 2:59
ADAPT JR Clinical Trial 5:15
Next Steps 8:47

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Robert Rapaport, MD, Professor of Pediatric Endocrinology, and Director of the Comprehensive Growth Center at the Icahn School of Medicine, Mount Sinai Medical Center, New York City, discusses the causes of growth hormone deficiency and its treatment. Growth failure in children is a considerable challenge for parents and pediatricians, with clinical and social stigma implications that may be avoided with early diagnosis.

The most important issue in young patients with growth failure is to detect it early, according to Dr. Rapaport. “As soon as you see a major deviation from the [expected growth chart] norm, act on it, even at age 2,” he emphasized, “because we know that best outcomes result from early detection.” A growth failure diagnosis is delayed or underdiagnosed in minority groups; it is underdiagnosed in girls relative to boys. In most cases, children are referred to the Comprehensive Growth Center by pediatricians and primary care physicians, and it should be monitored from birth. Growth failure in children can be caused by growth hormone (GH) deficiency, malnutrition, celiac disease, pituitary tumor (which suppresses the release of growth hormone) or a very rare genetic deletion. Once the potentially nonendocrine causes of GH deficiency are excluded, then causes related to the hypothalamus–pituitary-thyroid axis should be investigated, said Dr. Rapaport. 

Growth hormone stimulation testing and low blood levels of insulin-like growth factor (IGF) and IGF-binding protein concentrations can help confirm GH deficiency as the cause. However, low IGF-1 levels can also be caused by excessively high GH levels. In children diagnosed with GH deficiency, weekly GH injections are typically prescribed. In addition to monitoring these children for potential side effects of the GH injections, Dr. Rapoport recommended that they should undergo lab testing for IGF-1 blood concentrations every 3 to 6 months, until the bones fuse (signaling the conclusion of growth).

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Julia Scarisbrick, MD, MBhons, ChB, FRCP, is one of the leaders of the PROCLIPI (Prospective Cutaneous Lymphoma International Prognostic Index) study, a registry of patients with cutaneous T-cell lymphoma (CTCL). Dr. Scarisbrick, Professor of Dermatology, Department of Immunology and Immunotherapy, University of Birmingham, Birmingham, United Kingdom, discusses the study’s objectives, as well as challenges in the diagnosis of CTCL.

Cutaneous T-cell lymphoma (CTCL) is a rare group of skin malignancies that includes aggressive (e.g., Sézary syndrome) and nonaggressive or indolent forms (e.g, mycosis fungoides). However, advanced mycosis fungoides can be a dangerous cutaneous lymphoma.

One of the most important unmet needs in patients with CTCL has been differentiating patients at high risk for poor outcomes from those at lower risk, which can directly affect treatment choices.
The PROCLIPI (Prospective Cutaneous Lymphoma International Prognostic Index) trial, an international registry study, sought to use years of prospectively collected patient data (from 552 patients with advanced stage mycosis fungoides or Sézary syndrome) to develop a prognostic index. An implication of a successful new prognostic index would be an improvement in the existing disease staging algorithm (IA–IVB) to better reflect survival rates.

The use of this new prognostic index will allow physicians to identify the subset of patients at high risk for poor outcomes, which would directly affect treatment decision making.

Another important unmet need in CTCL is improving diagnosis. At present, diagnosis of CTCL depends on a combination of clinical findings, skin biopsies, and genetic tests. A physician who is evaluating a skin lesion may not include CTCL in the differential diagnosis, and as a result, the diagnosis delay can be 3 years or more. A singular diagnostic test would be extremely helpful for family doctors and community dermatologists in considering CTCL as a potential cause and potentially improve the diagnostic yield in the community. The data from this repository of 10 years of patient data may help lead to this diagnostic simplification. Diagnostic delay can result, over time, in T-cell cancer affecting the lymph nodes, blood, and even visceral organs.

CHAPTERS
Introduction 00:00
PROCLIPI Registry Insights 00:19
Reducing Diagnostic Challenges 2:38
Early Skin Symptoms 3:47
Implications on Patient Care 5:21
Impact on Diagnosis, Risk Stratification, and Decision Making 6:16
Take Home Message 7:14

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Carolina Barnett-Tapia, MD, Neuromuscular Neurologist and the University of Toronto, discusses the safety and efficacy of subcutaneous efgartigimod PH20 in ocular myasthenia gravis (oMG).

oMG is a neuromuscular disease characterized by autoantibody production against post-synaptic proteins in the neuromuscular junction. oMG typically presents as almost exclusively ocular symptoms and can evolve into generalized myasthenia gravis (gMG) in about 20% to 60% of cases. Common ocular manifestations include fluctuating ptosis, diplopia, and orbicularis weakness.

The Phase 3 ADAPT OCULUS trial (NCT06558279) is a randomized, double-blind, placebo-controlled, parallel-group design study evaluating the efficacy and safety of efgartigimod PH20 subcutaneous administered by a prefilled syringe in adults with oMG. Efgartigimod PH20 is a human immunoglobulin G1 (IgG1) antibody Fc fragment coformulated with recombinant human hyaluronidase PH20 that selectively reduces IgG levels by blocking neonatal Fc receptor–mediated IgG recycling.

In Part A of this trial, adults with confirmed oMG and a Myasthenia Gravis Impairment Index (MGII) patient-reported outcome subcomponent ocular score of 6 or greater are randomized to receive 4 once-weekly efgartigimod PH20 1000 mg or placebo, followed by 4 weeks of follow-up.

The primary endpoint is change in MGII patient-reported outcome ocular score from baseline to week 4. Key secondary endpoints include changes from baseline to week 4 in MGII ocular score (patient-reported outcome plus physical examination) and MGII total score. Safety assessments include adverse event incidence and severity. Topline results highlighted that the primary endpoint was met.

CHAPTERS
Introduction 00:00
Ocular Myasthenia Gravis 00:13
Susceptibility of Ocular Manifestations 1:53
ADAPT OCULUS Clinical Trial 3:05
Access to Treatment 5:43
Patient Burden 6:48

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Christopher Romero, MD, a pediatric endocrinologist at Mount Sinai Medical Center, New York City, and Associate Professor of Pediatrics at the Icahn School of Medicine at Mount Sinai discusses arginine vasopressin deficiency. The name of the rare disease central diabetes insipidus was changed in 2024 to better reflect its etiology.

Central diabetes insipidus, a rare disease, is unrelated to the common medical problem diabetes mellitus, other than they are both problems related to endocrinologic dysfunction. Whereas diabetes mellitus involves pancreatic function and the production of the hormone insulin, central diabetes insipidus involves the pituitary gland and regulation of the hormone vasopressin. Dr. Romero stated that a new name for central diabetes insipidus was introduced in 2024—arginine vasopressin deficiency (AVP-D) to reflect the difference and relieve misconceptions caused by the traditional naming. 

The central issue with AVP-D is the function of antidiuretic hormone, which regulates water concentrations in the body. Pediatric and adult patients with this vasopressin deficiency (which mediates antidiuretic hormone levels) excrete more urine than patients without the deficiency. “It causes these patients to drink more, to make up for the water loss,” said Dr. Romero, “resulting in kids being thirstier and having to use the bathroom more often.” 

As a result, AVP-D can lead to weight loss and loss of appetite, dehydration, and electrolyte abnormalities. He also pointed out that the abnormal cycle of drinking and urination in children interferes with school work and performance. 

“Unless you’re aware of [AVP-D], you may miss the diagnosis,” said Dr. Romero. The pituitary gland is involved with so many functions, and symptoms only slowly evolve. Issues with the onset of puberty and growth may hint at the pituitary source of the problem. 

Historically, treatment was managed with an oral formulation of vasopressin, which was first available in the 1970s. An intravenous form was available in inpatient settings. A nasal spray formulation was subsequently developed, and is useful particularly with older children. Dr. Romero pointed out, figuring out the correct dosage for an individual pediatric patient is key; every child with AVP-D is different in terms of how much water they lose during the drinking–urination cycle. “Even though the oral form was effective, only two dosages were available. You have to titrate the dose to balance the water loss,” he emphasized.

The introduction of Desmoda in February 2026, an oral solution of desmopressin acetate 0.05 mg/mL, allows for easier titration. The solution may be easier to take than the pills for young children, and caregivers may have a better idea of precisely how much medication the patient is getting. For those reasons, Dr. Romero believes this formulation may be the best option for young pediatric patients with AVP-D.
 

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Robert Rapaport, MD, Professor of Pediatric Endocrinology, and Director of the Comprehensive Growth Center at the Icahn School of Medicine, Mount Sinai Medical Center, New York City, discusses the causes of growth hormone deficiency and its treatment.

Growth failure in children is a considerable challenge for parents and pediatricians, with clinical and social stigma implications that may be avoided with early diagnosis.

The most important issue in young patients with growth failure is to detect it early, according to Dr. Rapaport. “As soon as you see a major deviation from the [expected growth chart] norm, act on it, even at age 2,” he emphasized, “because we know that best outcomes result from early detection.”

A growth failure diagnosis is delayed or underdiagnosed in minority groups; it is underdiagnosed in girls relative to boys. In most cases, children are referred to the Comprehensive Growth Center by pediatricians and primary care physicians, and it should be monitored from birth.

Growth failure in children can be caused by growth hormone (GH) deficiency, malnutrition, celiac disease, pituitary tumor (which suppresses the release of growth hormone) or a very rare genetic deletion. Once the potentially nonendocrine causes of GH deficiency are excluded, then causes related to the hypothalamus–pituitary-thyroid axis should be investigated, said Dr. Rapaport.

Growth hormone stimulation testing and low blood levels of insulin-like growth factor (IGF) and IGF-binding protein concentrations can help confirm GH deficiency as the cause. However, low IGF-1 levels can also be caused by excessively high GH levels.

In children diagnosed with GH deficiency, weekly GH injections are typically prescribed. In addition to monitoring these children for potential side effects of the GH injections, Dr. Rapoport recommended that they should undergo lab testing for IGF-1 blood concentrations every 3 to 6 months, until the bones fuse (signaling the conclusion of growth).

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Robert Rapaport, MD, Professor of Pediatric Endocrinology, and Director of the Comprehensive Growth Center at the Icahn School of Medicine, Mount Sinai Medical Center, New York City, discusses the causes of growth hormone deficiency and its treatment.

Growth failure in children is a considerable challenge for parents and pediatricians, with clinical and social stigma implications that may be avoided with early diagnosis.

The most important issue in young patients with growth failure is to detect it early, according to Dr. Rapaport. “As soon as you see a major deviation from the [expected growth chart] norm, act on it, even at age 2,” he emphasized, “because we know that best outcomes result from early detection.”

A growth failure diagnosis is delayed or underdiagnosed in minority groups; it is underdiagnosed in girls relative to boys. In most cases, children are referred to the Comprehensive Growth Center by pediatricians and primary care physicians, and it should be monitored from birth.

Growth failure in children can be caused by growth hormone (GH) deficiency, malnutrition, celiac disease, pituitary tumor (which suppresses the release of growth hormone) or a very rare genetic deletion. Once the potentially nonendocrine causes of GH deficiency are excluded, then causes related to the hypothalamus–pituitary-thyroid axis should be investigated, said Dr. Rapaport.

Growth hormone stimulation testing and low blood levels of insulin-like growth factor (IGF) and IGF-binding protein concentrations can help confirm GH deficiency as the cause. However, low IGF-1 levels can also be caused by excessively high GH levels.

In children diagnosed with GH deficiency, weekly GH injections are typically prescribed. In addition to monitoring these children for potential side effects of the GH injections, Dr. Rapoport recommended that they should undergo lab testing for IGF-1 blood concentrations every 3 to 6 months, until the bones fuse (signaling the conclusion of growth).

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