אמיפמפרידין
הערות שוליים

↑  Argov Z (Oct 2009). "Management of myasthenic conditions: nonimmune issues". Current Opinion in Neurology. 22 (5): 493–7. doi:10.1097/WCO.0b013e32832f15fa. PMID 19593127
↑  Angela Abicht, MD, Juliane Müller, S, PhD, and Hanns Lochmüller, MD. Congenital Myasthenic Syndromes  GeneReviews. University of Washington, Seattle.
1 2 3 4 5 6  Firdapse Summary of Product Characteristics  EMA. February 11, 2010
1 2  Keogh M, Sedehizadeh S, Maddison P (2011). "Treatment for Lambert-Eaton myasthenic syndrome". The Cochrane Database of Systematic Reviews (2): CD003279. doi:10.1002/14651858.CD003279.pub3. PMID 21328260.
↑  Lambert Eaton myasthenic syndrome
1 2  Tyler B. Tarr, Peter Wipf, and Stephen D. Meriney Synaptic pathophysiology and treatment of Lambert-Eaton myasthenic syndromeMolecular Neurobiology. 52 (1): 456–63. doi:10.1007/s12035-014-8887-2. PMC 4362862. PMID 25195700.
↑  G E Kirsch and T Narahashi 3,4-diaminopyridine. A potent new potassium channel blocker. Biophys J. 22 (3): 507–12. doi:10.1016/s0006-3495(78)85503-9. PMC 1473482. PMID 667299.
1 2  Dinnendahl, V; Fricke, U, eds. (2015). Arzneistoff-Profile (in German). 1 (28th ed.). Eschborn, Germany: Govi Pharmazeutischer Verlag. ISBN 978-3-7741-9846-3.
↑  Mahé, Nathalie; Nicolaï, Béatrice; Allouchi, Hassan; Barrio, Maria; Do, Bernard; Céolin, René; Tamarit, Josep-Lluis; Rietveld, Ivo B. (2013). "Crystal Structure and Solid-State Properties of 3,4-Diaminopyridine Dihydrogen Phosphate and Their Comparison with Other Diaminopyridine Salts". Cryst Growth Des. 13 (2): 708–715. doi:10.1021/cg3014249.
↑  A. Klement (9 November 2015). "Firdapse". Österreichische Apothekerzeitung (in German) (23/2015): 10f.
1 2  Evidence Review: Amifampridine phosphate for the treatment of LambertEaston Myasthenic Syndrome
1 2  Sabrina Tavernise F.D.A. Deals Setback to Catalyst in Race for Drug Approval New York Times.
↑  Drummond, M; Towse, A (May 2014). "Orphan drugs policies: a suitable case for treatment". The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care. 15 (4): 335–40. doi:10.1007/s10198-014-0560-1. PMID 24435513.
↑  Derek Lowe Catalyst Pharmaceuticals And Their Business Plan
1 2 3  Dalia Deak Jacobus and Catalyst Continue to Race for Approval of LEMS Drug  Bill of Health.
1 2  ED SILVERMAN A family-run drug maker faces tough choices to stay afloat in the Shkreli era STAT News.
1 2  JACOBUS PHARMACEUTICALS Catalyst Pharmaceutical shares plummet on competitor’s positive resultsDrug R&D Insight. 25 April, 2015.
1 2 3  Public summary of opinion on orphan designation 3,4 diaminopyridine phosphate for the for the treatment of Lambert-Eaton myasthenic syndrome  EMA.
↑  OPi ou comment construire une biopharma en moins de dix ans
1 2  ASSESSMENT REPORT FOR Zenas  EMA CHMP committee. 2009
↑  "Huxley Acquisition Lands Biomarin New LEMS Treatment" Pharmaceutical Technology. דורש רישום.
1 2  Ron Leuty BioMarin licenses North American rights to rare disease drug, invests $5M in Florida company BioMarin licenses North American rights to rare disease drug, invests $5M in Florida company
↑  Adrian Goldberg Drug firms accused of exploiting loophole for profit BBC News.
↑  Open letter to prime minister David Cameron and health secretary Andrew Lansley BMJ. 341: c6466. doi:10.1136/bmj.c6466. PMID 21081599.
↑  Hawkes N, Cohen D (2010). "What makes an orphan drug?". BMJ. 341: c6459. doi:10.1136/bmj.c6459. PMID 21081607.
↑  Vedeler, CA; Antoine, JC; Giometto, B; Graus, F; Grisold, W; Hart, IK; Honnorat, J; Sillevis Smitt, PA; Verschuuren, JJ; Voltz, R; Paraneoplastic Neurological Syndrome Euronetwork. (July 2006). "Management of paraneoplastic neurological syndromes: report of an EFNS Task Force". European Journal of Neurology. 13 (7): 682–90. doi:10.1111/j.1468-1331.2006.01266.x. PMID 16834698.
↑  International Nonproprietary Names for Pharmaceutical Substances (INN)
↑  Search Orphan Drug Designations and Approvals
↑  Danial E. Baker Breakthrough Drug Approval Process and Postmarketing ADR Reporting Hospital Pharmacy. 48 (10): 796–8. doi:10.1310/hpj4810-796. PMC 3859287. PMID 24421428.
↑  Search Orphan Drug Designations and Approvals FDA
↑  Search Orphan Drug Designations and Approvals www.accessdata.fda.gov. FDA
↑  Brian Bandell  Shares of Catalyst Pharmaceuticals soar 42% on FDA news South Florida Business Journal.
↑  James Radke Catalyst Using the Expanded Access Program to Conduct Phase IV Study with LEMS Patients Rare Disease Report
↑  Search Orphan Drug Designations and Approvals U.S. Food and Drug Administration
↑  Celia Ampel  Catalyst Pharma stock rises 5% after firm hits FDA milestone South Florida Business Journal.
↑  Burns, TM, et al.I (February 2016). "Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine". Muscle & Nerve. 53 (2): 165–8. doi:10.1002/mus.25009. PMID 26662952.
↑  Point d’information sur l’utilisation de la 3.4 diaminopyridine
↑  Sabrina Tavernise Patients Fear Spike in Price of Old Drugs New York Times
↑  Ben Adams Catalyst Pharmaceuticals hit by FDA extra studies request for Firdapse FierceBiotech
↑  Ben Adams Catalyst to ax 30% of workforce in wake of FDA trial demands FierceBiotech
↑  Debora Lima  Catalyst Pharmaceuticals files new drug application with FDA South Florida Business Journa.
↑  J.Stewart BPharm Firdapse Approval History Drugs.com
↑  Bernie Sanders asks why drug, once free, now costs $375,000 NBC News
↑  Family outraged over life-changing treatment going from free to $375,000 a year NBC News
↑  Solari, A.; Uitdehaag, B.; Giuliani, G.; Pucci, E.; Taus, C. (2002). "Aminopyridines for symptomatic treatment in multiple sclerosis". The Cochrane Database of Systematic Reviews (4): CD001330. doi:10.1002/14651858.CD001330. ISSN 1469-493X. PMID 12804404.
↑  Sedehizadeh, S; Keogh, M; Maddison, P (2012). "The use of aminopyridines in neurological disorders". Clinical Neuropharmacology. 35 (4): 191–200. doi:10.1097/WNF.0b013e31825a68c5. PMID 22805230.
↑  פירדפסה, עלון לרופא, משרד הבריאות
N-(4-Amino-3-pyridinyl)acetamide or 3-N-acetylamifampridine, the metabolite