Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma, Angelini Group - LARVOL DELTA

Decrement on Low-Frequency Repetitive Nerve Stimulation Is Not Synonymous With Myasthenia Gravis. (PubMed, Cureus) - "Amifampridine was subsequently initiated, followed by intravenous immunoglobulin, after which the patient demonstrated marked improvement in lower-limb strength and walking ability. Initial malignancy screening was negative, and longitudinal surveillance was planned. This case emphasizes that a decrement on low-frequency stimulation is not specific for myasthenia gravis and that low resting motor amplitudes with marked post-exercise facilitation strongly indicate a presynaptic neuromuscular junction disorder."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Myositis • Oncology • Pain • Xerostomia

Beyond the Neuromuscular Junction: Navigating Neuromuscular Fragility in Robotic Surgery (ASA 2026) - "She was chronically ventilator-dependent on home nasal BiPAP and treated with amifampridine and ephedrine. Anesthetic management was challenged by neuromuscular junction sensitivity to sedatives and neuromuscular blockers, prolonged postoperative ventilation, altered autonomic response, positioning and insufflation effects during robotic surgery, and the need to balance renal allograft perfusion with hemodynamic stability. This case highlights complexities in managing neuromuscular disease, chronic ventilatory dependence, and transplant physiology during advanced surgical procedures."

Surgery • Chronic Kidney Disease • CNS Disorders • Obstructive Sleep Apnea • Respiratory Diseases • Sleep Disorder

Perioperative Management of Pierson Syndrome and Congenital Myasthenic Syndrome (ASA 2026) - "Despite preserved baseline ambulation on amifampridine and ephedrine, anticipated perioperative respiratory weakness prompted planned postoperative ventilation while neuromuscular therapy was optimized...Her course was further complicated by acute upper extremity ischemia after arterial line removal, necessitating emergent thrombectomy and fasciotomies. This case highlights perioperative management in a rare multisystem disorder where syndromic complexity, ventilator dependence, and reintubation risk complicated routine postoperative planning."

Cardiovascular • Nephrology

Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS. (PubMed, J Neuromuscul Dis) - "While amifampridine is the established first-line symptomatic treatment, longitudinal data regarding the evolution of therapeutic regimens over time and the use of immunosuppressive therapies remain limited...Immunosuppressive drug use-predominantly prednisolone and azathioprine -increased over time, reaching 44% after five years...Immunomodulatory therapies were introduced gradually and their use increased over time. Compared with autoimmune myasthenia gravis, immunomodulatory therapy was used less frequently during the early disease course of LEMS."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Oncology

Two Skin Diseases, One Checkpoint Inhibitor: Cutaneous Toxicity From Pembrolizumab (BAD 2026) - "A 55-year-old man with locally advanced lung adenocarcinoma and previous chronic plaque psoriasis completed three cycles of neoadjuvant pembrolizumab–pemetrexed– carboplatin...The blistering increased and, after discussion with oncology, doxycycline was initiated and prednisolone weaned...BI13 (P038) Guselkumab for pembrolizumab-induced psoriasis in a patient with metastatic melanoma Olwyn Conlon, Maeve Herlihy, Yasmine Safta, Marina O’Kane, Eilis Ni Chinneide, Helen Barrett and Adrian Murphy This abstract is also presented as a Bristol Cup poster, P038, and is available at https://doi.org/10.1093/bjd/ljag086.065. Abstracts i179 BI14 (P162) Risks of tuberculosis, meningitis, candidiasis and fungal infections among patients receiving systemic treatments for psoriasis Heber Rew Bright Bright, 1 Catherine Smith, 2 Philip Laws, 3 Nick Reynolds, 4 Duc Binh Phan, 1 Mark Lunt, 1 Richard B Warren1 and Zenas Yiu1 This abstract is also presented as a Bristol Cup poster, P162, and..."

Checkpoint inhibition • Bullous Pemphigoid • Candidiasis • CNS Disorders • Dermatology • Dermatopathology • Immunology • Infectious Disease • Lung Adenocarcinoma • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Plaque Psoriasis • Psoriasis • Respiratory Diseases • Solid Tumor • Tuberculosis

Review of Congenital Myasthenic Syndrome Caused by Pathogenic Variants in GFPT1. (PubMed, Muscle Nerve) - "Pyridostigmine, amifampridine, and salbutamol were effective in 95.8%, 76.3%, and 86.4%, respectively...In mouse models, complete lack of GFPT1 in skeletal muscle developed CMS in 6 weeks of age, whereas lack of GFPT1-L in skeletal muscle required 12 months to develop CMS, which was likely to be accounted for by the expression of a low level of GFPT1-S. In accordance with this notion, null variants were enriched in exon 9 in GFPT1-CMS."

Journal • Review

INTEGRATING IVIG IN HOSPICE FOR NEUROLOGICAL PALLIATION: A COLLABORATIVE MODEL AND SCOPING REVIEW (MASCC-ISOO 2026) - "Despite being bed-bound and tube-fed, IVIG was started in June 2025 (with prednisolone and amifampridine) which improved alertness and upper limbs strength...Conclusions This is the first reported model enabling IVIG for neurological palliation within hospice, addressing a literature gap. It demonstrates that selected immunomodulatory therapies can be integrated into goal-concordant end-of-life care through structured collaboration and net benefit assessment, challenging the paradigm that hospice requires cessation of all disease-modifying treatments."

Review • CNS Disorders • Hematological Malignancies • Lung Cancer • Ophthalmology • Small Cell Lung Cancer • Solid Tumor

Lambert-Eaton myasthenic syndrome in United States veterans with small cell lung carcinoma (SCLC). (ASCO 2026) - "Among US veterans with SCLC, LEMS is a rare disorder consistently observed over the past 25 years with a slight negative, non-significant incidence trend. The majority of LEMS patients were treated with pyridostigmine rather than amifampridine. SCLC patients with LEMS exhibited improved survival compared to those with SCLC alone."

Hematological Disorders • Immunology • Lung Cancer • Oncology • Rare Diseases • Small Cell Lung Cancer • Solid Tumor

Obexelimab for the Treatment of IgG4-Related Disease. (PubMed, N Engl J Med) - P3 | "Among patients with active IgG4-related disease, weekly obexelimab treatment led to a significantly lower risk of disease flare and significantly less glucocorticoid exposure than placebo. (Funded by Zenas BioPharma; INDIGO ClinicalTrials.gov number, NCT05662241.)."

Journal • Immunology • Inflammation • Musculoskeletal Pain

HSR26-256: Real-World Evidence of Predictors of Time to Amifampridine Discontinuation and Survival in Patients With Cancer-Associated and Non-Cancer-Associated Lambert-Eaton Myasthenic Syndrome in the United States. (PubMed, J Natl Compr Canc Netw) - No abstract available

HEOR • Journal • Real-world evidence • Oncology

Efficacy and Safety of Amifampridine in Myasthenia Gravis: A Randomized, Double-Blind, Placebo-Controlled Crossover Trial. (PubMed, Neurology) - P3, P3/4 | "The addition of amifampridine to pyridostigmine was not superior to treatment with pyridostigmine alone and was associated with a higher incidence of adverse events."

Clinical • Journal • CNS Disorders • Fatigue • Myasthenia Gravis • Sleep Disorder

HSR26-256: Real-World Evidence of Predictors of Time to Amifampridine Discontinuation and Survival in Patients With Cancer-Associated and Non-Cancer-Associated Lambert-Eaton Myasthenic Syndrome in the United States. (PubMed, J Natl Compr Canc Netw) - No abstract available

HEOR • Journal • Real-world evidence • Oncology

Real-World Evidence of Predictors of Time to Amifampridine Discontinuation and Survival in Patients With Cancer–Associated and Non-Cancer–Associated Lambert-Eaton Myasthenic Syndrome in the United States (NCCN 2026) - "Among patients with LEMS receiving amifampridine, the risk of treatment discontinuation or death was significantly higher in patients who were older (>63yrs), have a cancer diagnosis, not dose titrated, and prescribed amifampridine at a dose less than 52 mg/d. Given the limitations of this retrospective analysis, these findings warrant additional review as the use of amifampridine increases. View Full Size HSR26-256: FIGURE."

Clinical • HEOR • Real-world • Real-world evidence • Immunology • Oncology

PharmVar GeneFocus: NAT2-Genetic Variation and Updated Nomenclature. (PubMed, Clin Pharmacol Ther) - "NAT2 metabolizes several clinically used drugs including isoniazid, hydralazine, amifampridine, procainamide, and sulfonamides such as dapsone, and also some highly carcinogenic arylamines. We also highlight and discuss challenges regarding the characterization of allelic variation and determination of allele frequencies across world populations. The "new" NAT2 PharmVar nomenclature is utilized by ClinPGx (formerly PharmGKB) and the Clinical Pharmacogenetics Implementation Consortium (CPIC)."

Journal • Review • NAT2

To Treat or Not To Treat - Immune Checkpoint Inhibition in Patients with SCLC-associated LEMS (SNO 2025) - "He was started on carboplatin, etoposide, and atezolizumab after which his weakness and diplopia worsened...One year later, she was found to have brain metastasis treated with stereotactic radiosurgery and durvalumab... Additional data is needed to better understand the safety of ICI in SCLC-associated LEMS."

Checkpoint inhibition • Clinical • CNS Disorders • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor

To Treat or Not To Treat - Immune Checkpoint Inhibition in Patients with SCLC-associated LEMS (WFNOS 2025) - "He was started on carboplatin, etoposide, and atezolizumab after which his weakness and diplopia worsened...One year later, she was found to have brain metastasis treated with stereotactic radiosurgery and durvalumab... Additional data is needed to better understand the safety of ICI in SCLC-associated LEMS."

Checkpoint inhibition • Clinical • CNS Disorders • Lung Cancer • Small Cell Lung Cancer • Solid Tumor

UNMASKING LEMS: A CASE OF LAMBERT-EATON MYASTHENIC SYNDROME IN THE CONTEXT OF MULTIPLE SCLEROSIS (CHEST 2025) - "The patient initiated radiation with adjuvant chemotherapy of carboplatin and etoposide...First-line treatment consists of cancer-directed treatment, if indicated, as well as the potassium-channel blocker amifampridine phosphate, which serves to enhance acetylcholine release at the neuromuscular junction. This case highlights the importance of careful neurologic examination for consideration of LEMS in patients with underlying neurologic conditions. Early recognition and appropriate management, including treatment of possible underlying malignancy such as SCLC, are critical to improving patient outcomes in this population."

Clinical • CNS Disorders • Cough • Gastrointestinal Disorder • Lung Cancer • Multiple Sclerosis • Pulmonary Disease • Respiratory Diseases • Small Cell Lung Cancer • Solid Tumor • Xerostomia • CRP

Exploring a gap of the 2024 McDonald criteria: the role of two spinal cord lesions plus oligoclonal bands, and transverse spinal cord lesion extent, in patients with a typical attack onset (ECTRIMS 2025) - "Luca Bollo has received a Rio Hortega grant, from the Instituto de Salud CarlosIII, Spain (CM24/XXX) and a one-year stipend endowed by the NMSS/AAN John Dystel Prize for Multiple Sclerosis Research awarded to Prof...Xavier Montalban's institution has received compensation for lecture honoraria and travel expenses, participation in scientific meetings, clinical trial steering committee membership, or clinical advisory board participation in recent years from Abbvie, Actelion, Alexion, Astrazeneca, Autolus, Bial PD, Biogen, Bristol-Myers Squibb/Celgene, EMD Serono, Genzyme, Hoffmann-La Roche, Immunic Therapeutics, Indivi, Janssen Pharmaceuticals,Juvisé Pharmaceutical, Lilly, Medday, Medscape, Merck, Mylan-Viatris, Nervgen, Neuraxpharm, Novartis, Peervoice, Rewind Therapeutics, Samsung-Biosys, Sandoz, Sanofi-Genzyme, Teva Pharmaceutical, TG Therapeutics, Zenas Biopharma, Excemed, ECTRIMS, MSIF, and NMSS or any of their affiliates... A transverse lesion extent =2 SC..."

Clinical • Late-breaking abstract • CNS Disorders • Multiple Sclerosis

Exploring early RAW and early PIRA as definitions of aggressive multiple sclerosis and as combined predictors of confirmed EDSS 4.0 at 5 years (ECTRIMS 2025) - "Luca Bollo has received a Rio Hortega grant, from the Instituto de Salud CarlosIII, Spain (CM24/XXX) and a one-year stipend endowed by the NMSS/AAN John Dystel Prize for Multiple Sclerosis Research awarded to Prof...Xavier Montalban's institution has received compensation for lecture honoraria and travel expenses, participation in scientific meetings, clinical trial steering committee membership, or clinical advisory board participation in recent years from Abbvie, Actelion, Alexion, Astrazeneca, Autolus, Bial PD, Biogen, Bristol-Myers Squibb/Celgene, EMD Serono, Genzyme, Hoffmann-La Roche, Immunic Therapeutics, Indivi, Janssen Pharmaceuticals,Juvisé Pharmaceutical, Lilly, Medday, Medscape, Merck, Mylan-Viatris, Nervgen, Neuraxpharm, Novartis, Peervoice, Rewind Therapeutics, Samsung-Biosys, Sandoz, Sanofi-Genzyme, Teva Pharmaceutical, TG Therapeutics, Zenas Biopharma, Excemed, ECTRIMS, MSIF, and NMSS or any of their affiliates... We found associations of older..."

Late-breaking abstract • CNS Disorders • Multiple Sclerosis

Firdapse for Post-BOTOX Vocal Weakness (clinicaltrials.gov) - P2 | N=18 | Completed | Sponsor: Augusta University | N=10 ➔ 18 | Trial completion date: Dec 2023 ➔ Sep 2025 | Trial primary completion date: Dec 2023 ➔ May 2025 | Recruiting ➔ Completed

Enrollment change • Trial completion • Trial completion date • Trial primary completion date • Dysphonia

Diagnostic Performance of Paramagnetic Rim Lesions in Multiple Sclerosis in a Large, Prospective, Multicenter Cohort: Insights from the CAVS-MS Study (ECTRIMS 2025) - "Christina J Azevedo: Received consulting fees from Genentech, Zenas Biopharma, EMD Serono, Alexion Pharmaceuticals, Sanofi Genzyme... Our findings from the largest, prospective, international study on diagnostic performance of PRL in MS support the inclusion of PRL in the 2024 McDonald Criteria by demonstrating PRL as a broadly applicable imaging biomarker with high diagnostic specificity (particularly if multiple) and sensitivity (as a potential substitute for dissemination in space). We anticipate that sensitivity and specificity of baseline PRL will increase with ongoing follow-up of the CAVS-MS cohort, which will also enable definitive validation of baseline PRL as a valuable diagnostic biomarker. Disclousure of interest: Joshua D Lee: Nothing to disclose."

Clinical • Late-breaking abstract • CNS Disorders • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Multiple Sclerosis • Rheumatology

A Phase 2, Open Label, Sequential Group, Investigator Blinded Study ofMagnetic Resonance Spectroscopy (to Assess the Effects ofCNM-Au8 for the Bioenergetic Improvement of Impaired Neuronal RedoxState in Multiple Sclerosis (REPAIR-MS) - Results (ECTRIMS 2025) - " REPAIR-MS cohort 1 included relapsing remitting MS, stable on natalizumab...Sguigna received personal compensation for consulting services from Genentech, EMD Serono, Horizon Therapeutics, Amgen, and Bristol Myers Squibb and has received research support from Genentech, Clene Nanomedicine, Zenas Biopharma, IPMSA through the NMSS, PCORI, DOD/CDMRP, and NIH/NIA through his institution... CNM-Au8 showed evidence of improved brain bioenergetics improvement in MS participants with an average percent increase in the NAD+/NADH ratio of 9.5% (p=0.0275). CNM-Au8 treatment was safe and well-tolerated with treatment emergent adverse events predominantly assessed as mild-to-moderate severity, and transient. This data buttresses the theory that bioenergetic failure contributes to neurodegeneration in MS."

Clinical • Late-breaking abstract • P2 data • CNS Disorders • Movement Disorders • Multiple Sclerosis • Parkinson's Disease

AGNOS: A Phase 4 Study of Ofatumumab in Treatment-Naïve Participants with Very Early Relapsing-Remitting Multiple Sclerosis Compared with Healthy Controls (ECTRIMS 2025) - "Darin T Okuda has received personal compensation for consulting and advisory services from Biogen Inc., Cortechs.AI, Eisai, EMD Serono, Genentech, Genzyme/Sanofi, Immunic Therapeutics, Moderna, Octave Bioscience, Pfizer, and Zenas BioPharma along with research support from Alexion and Novartis... This pre-planned analysis confirms the efficacy of OMB in participants with very early, recently diagnosed, treatment-naïve RRMS in the AGNOS study, with over 80% of participants achieving NEDA-3 from months 6 to18. Functional performance and imaging outcomes in OMB-treated participants appeared broadly similar to those observed in healthy controls up to 18 months. Safety findings were consistent with prior reports."

Clinical • Late-breaking abstract • P4 data • CNS Disorders • Fatigue • Infectious Disease • Multiple Sclerosis • Musculoskeletal Pain • Novel Coronavirus Disease

Epidemiological analysis of Lambert-Eaton myasthenic syndrome in Türkiye: insights from a nationwide electronic health database. (PubMed, Front Neurol) - "In this study, we aimed to determine the epidemiological profile of LEMS in Türkiye, and to assess associated malignancies, mortality, and prescription rates of pyridostigmine and amifampridine...This is the first nationwide epidemiological study of LEMS in Türkiye, revealing lower incidence and prevalence rates than in other countries. This study provides valuable large-scale epidemiological data, enriching the global understanding of this rare disorder."

Journal • Immunology • Lung Cancer • Oncology • Rare Diseases • Small Cell Lung Cancer • Solid Tumor

Lambert Eaton Myasthenic Syndrome. (PubMed, Int Rev Neurobiol) - "Treatment of LEMS comprises symptomatic treatments such as 3,4 diaminopyridine (amifampridine), which increases quantal release; immunotherapy; and treatment of underlying malignancy if present. The life expectancy of non-tumour LEMS is normal, although complete remission is uncommon. Progression of SCLC determines prognosis in tumour-associated LEMS, which is nonetheless better than in SCLC without LEMS."

Journal • Review • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor • Xerostomia