dichlorphenamide / Generic mfg. - LARVOL DELTA

Safety and efficacy of dichlorphenamide in patients with periodic paralysis: A systematic review and meta-analysis. (PubMed, Neuromuscul Disord) - "Along with therapeutic benefits, some adverse effects were also associated with dichlorphenamide, such as paresthesias (p < 0.00001), cognitive disturbances (p: 0.008), dysgeusia (p: 0.01), and rash (p: 0.006). The GRADE analysis confirmed high-certainty evidence for primary outcomes, supporting dichlorphenamide's consistent efficacy across etiologies and ages, though its side effects necessitate careful monitoring and counseling."

Journal • Retrospective data • Review • CNS Disorders • Cognitive Disorders

SOMETIMES LESS IS NOT MORE: A CASE OF HYPOKALEMIC CARDIAC ARREST IN A HYPERKALEMIC PERIODIC PARALYSIS PATIENT (CHEST 2025) - "Given the limited knowledge of these genetic disorders, they can present in various forms, so we must remain vigilant and prepared for diverse presentations."

Clinical • Cardiovascular • Genetic Disorders • Renal Disease

A Clinical Overlap Presentation of Episodic Ataxia Type 2 and Periodic Paralysis with a Novel Mutation in CACNA1A (MDS Congress 2025) - "We present a unique case of clinical overlap of EA2 and PP symptoms in a patient with a novel variant in CACNA1A, widening the existing clinical and genetic heterogeneity of EA2. This case also provides support for treatment of EA2 with dichlorphenamide, particularly in those with the CACNA1A (c.5216 T>C. P.(F1739S) mutation who do not initially respond to acetazolamide."

Clinical • Ataxia • CNS Disorders • Movement Disorders • CACNA1A

Systemic Carbonic Anhydrase Inhibitors in Common Ophthalmic Diseases: A Scoping Review from A Clinical Standpoint. (PubMed, Curr Ophthalmol Rep) - "It's important to be aware of potential side effects and ensure the correct usage of oral CAIs, especially in individuals with other systemic diseases. A tailored evaluation of risks and benefits carried out for each individual, particularly in case of prolonged usage, should decrease the risk of adverse events."

Journal • Review • Cardiovascular • CNS Disorders • Glaucoma • Hypertension • Macular Edema • Ophthalmology

High altitude renal syndrome: four elements or one source? (PubMed, Ren Fail) - "Carbonic anhydrase inhibitors (e.g., acetazolamide) reduce hematocrit and improve oxygen saturation, but newer agents like methazolamide and dichlorphenamide offer equivalent efficacy with fewer side effects (e.g., reduced central nervous system toxicity). Additional interventions include calcium channel blockers (nifedipine), urate-lowering drugs, and experimental therapies such as HIF-2α inhibitors (PT2385) and endothelin receptor antagonists (matitan). This analysis underscores HAPC as the primary etiology of HARS, advocating revised diagnostic criteria and treatment prioritization."

Journal • Review • Cardiovascular • Hematological Disorders • Hypertension • Nephrology • Renal Disease • EPAS1

Xeris Biopharma Delivers Record Fourth Quarter and Full-Year 2024 Results; and Announces 2025 Guidance (Businesswire) - "Gvoke: Net revenue was $82.8 million for the year ended December 31, 2024 – an increase of approximately 24% compared to prior year...Recorlev: Net revenue was $64.3 million for the year ended December 31, 2024 – an increase of approximately 118% compared to prior year...Keveyis: Net revenue was $49.5 million for the year ended December 31, 2024 – a decrease of approximately 13% compared to prior year."

Commercial • CNS Disorders • Cushing’s Disease • Endocrine Disorders • Severe Hypoglycemia • Type 2 Diabetes Mellitus

Semaglutide (S) Completely Reverses the Severe Chronic Myopathy of Hyperkalemic Periodic Paralysis (KIDNEY WEEK 2024) - "His father, uncle, sister, & 3 nephews have the same mutation.He was treated with acetazolamide, albuterol, & daranide without success.S was given due to a body mass index greater than 40. By altering insulin signaling and skeletal muscle physiology, S increases intracellular potassium storage and decreases myopathy in HPP. S is an excellent & novel once weekly option that treats not just the hyperkalemic periodic paralysis but also the skeletal muscle weakness in a multimodal way.SPPBSPPBPre S4 mo7 mo12 moSide by Side Test (sec/ score)4/029/1>30/1180/1Semi Tandem Gait Test (sec/ score)1/015/1>30/145/1Tandem Gait Test (sec/ score)0/020/2>20/215/23 Meter Standard Gait Test (sec/ score)10.1/04.25/32.87/43.52/43 Meter Fast Gait Test (sec/ score)6.8/03.0/41.8/43.25/4Repeat Chair Stand Test (sec/ #/ score)0/0/025.1/5/115.4/5/220.5/5/1Total Score (normal >12)0121413BMI (kg/m2)36.533.231.329.2"

Muscular Atrophy • Myositis

Identification, crystallization, and first X-ray structure analyses of phenyl boronic acid-based inhibitors of human carbonic anhydrase-II. (PubMed, Int J Biol Macromol) - "Therapeutically used CA inhibitors (CAI) are sulfonamide-based, such as acetazolamide, dichlorphenamide, methazolamide, ethoxzolamide, etc. However, they exhibit several undesirable effects such as numbness, tingling of extremities, malaise, metallic taste, fatigue, renal calculi, and metabolic acidosis. In addition, a bond of 3.11 Å between the zinc ion and coordinated boron atom of the boronic acid moiety of compound 1 was also observed, contributing to binding affinity of compound 1 for hCA II. PDB ID: 8IGF."

Journal • CNS Disorders • Epilepsy • Fatigue • Glaucoma • Metabolic Disorders • Ophthalmology • Renal Calculi • CA2

Recurrent CACNA1A Variant Identified in Patients Referred for Periodic Paralysis (MDA 2024) - "The identification of the F1739S variant in two patients with EA2 strongly suggests pathogenicity of this genetic finding. These cases highlight the need to consider EA2 in the diagnostic and genetic evaluation of patients with periodic loss of ambulation and abnormal long-exercise testing. Additional studies may help understand the overlap between these disorders."

Clinical • Ataxia • CNS Disorders • Epilepsy • Genetic Disorders • Migraine • Movement Disorders • Otorhinolaryngology • Pain • Vertigo • CACNA1A

Exploring bioactive compounds from a symbiotic bacterial strain of Spongiobacter sp. (PubMed, Bioinformation) - "Using LC-MS analysis bioactive compounds Dichlorphenamide, Amifloxacin and Carbenicillin are identified in both positive and negative mode...EA276. Our results highlighted the significance of marine flora inhabiting sponges from the Red Sea as potential source of bioactive compounds and plant growth hormones of biological and agricultural significance."

Journal

Torrent Pharma launches First and Only Generic Version of Keveyis (dichlorphenamide) Tablets in the United States (PRNewswire) - "Torrent Pharma has begun shipping the generic version of Xeris's Keveyis® (dichlorphenamide), 50mg tablets, following final approval from the U.S. Food and Drug Administration for its Abbreviated New Drug Application. Torrent is distributing the product through select specialty pharmacies beginning this month."

Generic launch • CNS Disorders

Pyrazole/pyrazoline as an excellent pharmacophore in the design of carbonic anhydrase inhibitors (2018-2022). (PubMed, Arch Pharm (Weinheim)) - "Some examples of approved CA inhibitors are dorzolamide, methazolamide, brinzolamide, and dichlorphenamide. Hence, research that took place in the field of drug design and discovery of CA inhibition has been systematically reviewed and collated. Alongside, the structure-activity relationship has been described, followed by a description of the most potent molecules and their structural features."

Journal • Review • CNS Disorders • Epilepsy • Genetic Disorders • Glaucoma • Obesity • Oncology • Ophthalmology

Drug repurposing in skeletal muscle ion channelopathies. (PubMed, Curr Opin Pharmacol) - "Thus, ongoing research aims at the identification of repurposed drugs alternative to mexiletine and dichlorphenamide to allow personalization of treatment. This review highlights how drug repurposing may represent an efficient strategy in rare diseases, allowing reduction of drug development time and costs in a context in which the return on investment may be particularly challenging."

Clinical • Journal • Review • Genetic Disorders • Myotonic Dystrophy • Rare Diseases

Neuroprotective Effects of Carbonic Anhydrase Inhibition and Cyclic Adenosine Monophosphate Activation in Mouse Model of Transient Global Cerebral Ischemia and Reperfusion. (PubMed, Neuromolecular Med) - "In this investigation, the potential of dichlorphenamide (CAI) and the role of cAMP against ischemia-reperfusion injury were evaluated using a transient global cerebral I/R (tGCI/R) model...However, DCPA and FSK in combination showed much enhanced therapeutic outcomes against tGCI/R. Therefore, CA and cAMP present novel targets that may retard the progress of a transient ischemic attack to a full-blown stroke."

Journal • Preclinical • Cardiovascular • CNS Disorders • Reperfusion Injury • Vascular Neurology

The role of nephrologists in management of hypokalemic periodic paralysis: a case report. (PubMed, J Med Case Rep) - "This case demonstrates the crucial role nephrologists can play, not only in the diagnosis of hypokalemic periodic paralysis, but also in the ongoing management of this condition. Patients should be advised to regularly follow up with their nephrology team for evaluation due to the risk of developing myopathy."

Journal • Myositis • Nephrology

Dichlorphenamide for Refractory Hyperkalemic Periodic Paralysis. (PubMed, J Clin Neuromuscul Dis) - No abstract available

Journal

Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis. (PubMed, Muscle Nerve) - P3 | "One year open-label DCP treatment following a 9-week randomized, controlled study confirmed long-term DCP remains safe and effective for chronic use. Tolerability issues (paresthesia, cognition-related AEs) were manageable in most patients. ClinicalTrials.gov identifier NCT00494507."

Clinical • Journal

Increased sarcolemma chloride conductance as one of the mechanisms of action of carbonic anhydrase inhibitors in muscle excitability disorders. (PubMed, Exp Neurol) - "To get insight into the mechanism of action of carbonic anhydrase inhibitors (CAI) in neuromuscular disorders, we investigated effects of dichlorphenamide (DCP) and acetazolamide (ACTZ) on ClC-1 chloride channels and skeletal muscle excitability. These results suggest that carbonic anhydrase inhibitors can reduce muscle excitability by increasing ClC-1 channel activity, probably through intracellular acidification. Such a mechanism may contribute in part to the clinical effects of these drugs in myotonia and other muscle excitability disorders."

Journal • CNS Disorders

[VIRTUAL] Clinical experience with Dichlorphenamide in Refractory Hyperkalemic Periodic Paralysis with SCN4 Gene Mutation. (AAN 2021) - "Acetazolamide was then added which reduced attack rate to 3 per week, lasting several hours. This study demonstrated successful response to dichlorphenamide in a patient with treatment refractory hyperkalemic periodic paralysis with SCN4 gene mutation with complete resolution of attacks for over 2 year time period."

Clinical • CNS Disorders

[VIRTUAL] Marked Reduction in Paralytic Attacks in Patient With Andersen-Tawil Syndrome Switched From Acetazolamide to Dichlorphenamide: A Case Report (AAN 2021) - "Arrhythmias are currently controlled by a regimen of imipramine and a beta-blocker; the patient also has an implantable cardioverter-defibrillator. In this 24-year-old woman with ATS, switching from AZM to DCP provided marked reduction in the frequency and severity of PP and notable improvements in functioning, with no observed adverse effects."

Clinical • CNS Disorders

A connectivity map-based drug repurposing study and integrative analysis of transcriptomic profiling of SARS-CoV-2 infection. (PubMed, Infect Genet Evol) - "The results suggest the effectiveness of lansoprazole, folic acid, sulfamonomethoxine, tolnaftate, diclofenamide, halcinonide, saquinavir, metronidazole, ebselen, lidocaine and benzocaine, histone deacetylase (HDAC) inhibitors, heat shock protein 90 (HSP90) inhibitors, and many other clinically approved drugs as potent drugs against COVID-19 outbreak. Making new drugs remain a lengthy process, so the drug repurposing approach provides an insight into the therapeutics that might be helpful in this pandemic. In this study, pathway enrichment and protein network analysis are also performed, and the effectiveness of some drugs obtained from the CMap analysis has been investigated according to previous researches."

Journal • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • HSP90AA1

Targeted Therapies for Skeletal Muscle Ion Channelopathies: Systematic Review and Steps Towards Precision Medicine. (PubMed, J Neuromuscul Dis) - "These studies provided limited information about the response to treatments of individual mutations or groups of mutations. A major effort is needed to perform human studies for designing a mutation-driven precision medicine in muscle ion channelopathies."

Clinical • Journal • Review • Myasthenia Gravis • Myositis • Myotonic Dystrophy

[VIRTUAL] Improvement of strength and muscle MRI with dichlorphenamide in hypokalemic periodic paralysis myopathy: A case report (WMS 2020) - No abstract available

Clinical • Myositis

Efficacy and Safety of Dichlorphenamide for Primary Periodic Paralysis in Adolescents Compared With Adults. (PubMed, Pediatr Neurol) - "Dichlorphenamide was comparably effective and tolerated among a small number of adolescents as well as adults, although types of adverse events differed between groups."

Clinical • Journal • Dermatology • Rare Diseases

Episodic Muscle Disorders. (PubMed, Continuum (Minneap Minn)) - "This article will help neurologists diagnose and manage episodic muscle disorders and, in particular, the periodic paralyses and the nondystrophic myotonias."

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