Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma 
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 9 Diseases   6 Trials   6 Trials   268 News 


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  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    The Burden of Multimorbidity in ANCA-Associated Vasculitis: A Cohort Study (Poster Hall; in person) -  Sep 23, 2023 - Abstract #ACRConvergence2023ACR_Convergence_3606;    
    MM in AAV is characterized by clusters defined by morbidity burdens that vary over disease course and reflect a high impact of disease and its treatment. MM in AAV is associated with mortality risk.
  • ||||||||||  Rinvoq (upadacitinib) / AbbVie, elsubrutinib/upadacitinib (ABBV-599) / AbbVie, elsubrutinib (ABBV-105) / AbbVie
    Efficacy and Safety of ABBV-599 High Dose (Elsubrutinib 60 mg and Upadacitinib 30 mg) and Upadacitinib Monotherapy for the Treatment of Systemic Lupus Erythematosus: A Phase 2, Double-blind, Placebo-controlled Trial (Ballroom 20D; in person) -  Sep 23, 2023 - Abstract #ACRConvergence2023ACR_Convergence_1985;    
    P2
    J. Merrill: AbbVie, 2, Alexion, 2, Alumis, 2, Amgen, 2, AstraZeneca, 2, 5, Aurinia, 2, Bristol Myers Squibb, 2, 5, EMD Serono, 2, Genentech, 2, Gilead, 2, GlaxoSmithKline, 2, 5, Lilly, 2, Merck, 2, Pfizer, 2, Provention, 2, Remegen, 2, Sanofi, 2, UCB Pharma, 2, Zenas, 2; Y. Tanaka: AbbVie, 6, AstraZeneca, 6, BMS, 6, Boehringer-Ingelheim, 6, Chugai, 5, 6, Eisai, 5, 6, Eli Lilly, 6, Gilead, 6, GSK, 6, Mitsubishi-Tanabe, 5, Pfizer, 6, Taiho, 6, Taisho, 5, 6; D. D'Cruz: Eli Lilly, 2, GlaxoSmithKlein(GSK), 2, UCB, 2; K. Vila-Rivera: AbbVie/Abbott, 2; D. Siri: AbbVie/Abbott, 5, Boehringer-Ingelheim, 5, Bristol-Myers Squibb(BMS), 5, Eli Lilly, 5, Gilead, 5, GlaxoSmithKlein(GSK), 5, Hoffman Laroche, 5, Janssen, 5, Sanofi, 5;
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma, Botox (onabotulinumtoxin A) / GSK, AbbVie, Healis Therap
    Trial completion date, Trial primary completion date:  Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA (clinicaltrials.gov) -  Jul 3, 2023   
    P1,  N=20, Not yet recruiting, 
    Furthermore, this entry criterion decreases the eligible patient pool by almost 40% significantly slowing recruitment rates. Trial completion date: Jan 2024 --> Apr 2024 | Trial primary completion date: Jan 2024 --> Apr 2024
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma, Botox (onabotulinumtoxin A) / GSK, AbbVie, Healis Therap
    Trial completion date, Trial primary completion date:  Firdapse for Post-BOTOX Vocal Weakness (clinicaltrials.gov) -  May 26, 2023   
    P2,  N=10, Recruiting, 
    Collectively, co-administration of acetaminophen with amifampridine may lead to relevant drug interactions; thus, care should be taken during co-administration. Trial completion date: Jun 2023 --> Dec 2023 | Trial primary completion date: Jun 2023 --> Dec 2023
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion date, Trial primary completion date:  Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) (clinicaltrials.gov) -  Apr 18, 2023   
    P3,  N=70, Active, not recruiting, 
    Trial completion date: Jun 2023 --> Dec 2023 | Trial primary completion date: Jun 2023 --> Dec 2023 Trial completion date: Oct 2022 --> Apr 2023 | Trial primary completion date: Sep 2022 --> Apr 2023
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Lambert-Eaton Myasthenic Syndrome and Botulism. (Pubmed Central) -  Dec 21, 2022   
    A targeted clinical history and a thorough neurologic examination with support from serologic and electrodiagnostic studies are key to early diagnosis of LEMS and botulism. Early diagnosis of both conditions creates opportunities for therapy and improves outcomes.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Review, Journal:  Amifampridine for Lambert-Eaton myasthenic syndrome. (Pubmed Central) -  Nov 18, 2022   
    Early diagnosis of both conditions creates opportunities for therapy and improves outcomes. No abstract available
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion date, Trial primary completion date:  Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) (clinicaltrials.gov) -  Jul 23, 2022   
    P3,  N=70, Active, not recruiting, 
    SMA-001 study provided Class I evidence that AP was safe and effective in treating ambulatory patients affected by SMA type 3. Trial completion date: Jun 2022 --> Oct 2022 | Trial primary completion date: May 2022 --> Sep 2022
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion date, Trial termination, Trial primary completion date:  SMA3: Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (clinicaltrials.gov) -  Mar 29, 2022   
    P2,  N=12, Terminated, 
    No new safety signals were observed following long-term management of LEMS with 3,4-DAPP. Trial completion date: Apr 2022 --> Sep 2021 | Enrolling by invitation --> Terminated | Trial primary completion date: Dec 2021 --> Sep 2021; Development of indication not being pursued
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial primary completion date:  Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) (clinicaltrials.gov) -  Mar 10, 2022   
    P3,  N=70, Active, not recruiting, 
    Trial completion date: Apr 2022 --> Sep 2021 | Enrolling by invitation --> Terminated | Trial primary completion date: Dec 2021 --> Sep 2021; Development of indication not being pursued Trial primary completion date: Dec 2021 --> May 2022
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Lambert-Eaton myasthenic syndrome in a young girl. (Pubmed Central) -  Mar 3, 2022   
    She received plasma exchange electrophoresis, intravenous immunoglobulin, oral steroids and amifampridine with some clinical improvement. We present a case of a child with a demonstrated autoimmune propensity and no evidence of malignancy, thereby adding to the 12 previously reported cases of LEMS in children.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Botox (onabotulinumtoxin A) / GSK, AbbVie, Healis Therap
    New P2 trial:  Firdapse for Post-BOTOX Vocal Weakness (clinicaltrials.gov) -  Nov 17, 2021   
    P2,  N=10, Recruiting, 
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    [VIRTUAL] A Family with an Autosomal Dominantly Inherited CHRNA1 Mutation and Congenital Myasthenia Syndrome () -  Oct 21, 2021 - Abstract #ANA2021ANA_423;    
    This mutation was previously reported in compound heterozygosity in a CMS cases however with care ful phenotyping and whole genome sequencing we deter mined this mutant as likely causative in heterozygosity. Additionally we show clinical response to 3,4DAPP in this phenotype, suggestive of a slow-channel physiology.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Lambert-Eaton myasthenic syndrome revealed after surgery of lung adenocarcinoma. (Pubmed Central) -  Oct 5, 2021   
    One month later patient present a muscle weakness in both lower limbs and fatigability followed by an inability to walk. The diagnosis of LEMS was made from the distinctive electromyogram (EMG) findings and a treatment with Amifampridine (3, 4-diaminopyridine phosphate [3, 4-DAP]) was prescribed with evident efficacy for symptoms.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Enrollment change:  Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) -  Aug 18, 2021   
    P3,  N=93, Completed, 
    The diagnosis of LEMS was made from the distinctive electromyogram (EMG) findings and a treatment with Amifampridine (3, 4-diaminopyridine phosphate [3, 4-DAP]) was prescribed with evident efficacy for symptoms. N=70 --> 93
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Thanks zenas! (Twitter) -  Jul 7, 2021   
  • ||||||||||  Analgin (metamizole) / Sedico, Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Quinamed (amonafide) / Teva
    Biomarker, Review, Journal:  Arylamine N-acetyltransferase acetylation polymorphisms: paradigm for pharmacogenomic-guided therapy- a focused review. (Pubmed Central) -  Apr 16, 2021   
    Studies in human subjects and cryopreserved human hepatocytes show evidence for rapid, intermediate and slow acetylator phenotypes, with further data suggesting genetic heterogeneity within the slow acetylator phenotype. Incorporation of more robust NAT2 genotype/phenotypes relationships, including genetic heterogeneity within the slow acetylator phenotype should lead to further advancements in both health outcomes and cost benefit for prevention and treatment of tuberculosis.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion date, Trial primary completion date:  SMA3: Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (clinicaltrials.gov) -  Feb 16, 2021   
    P2,  N=12, Enrolling by invitation, 
    Incorporation of more robust NAT2 genotype/phenotypes relationships, including genetic heterogeneity within the slow acetylator phenotype should lead to further advancements in both health outcomes and cost benefit for prevention and treatment of tuberculosis. Trial completion date: Apr 2021 --> Apr 2022 | Trial primary completion date: Dec 2020 --> Dec 2021
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion date, Trial primary completion date:  Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis) (clinicaltrials.gov) -  Feb 16, 2021   
    P3,  N=70, Active, not recruiting, 
    Trial completion date: Apr 2021 --> Apr 2022 | Trial primary completion date: Dec 2020 --> Dec 2021 Trial completion date: Sep 2021 --> Jun 2022 | Trial primary completion date: Jun 2021 --> Dec 2021
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Amifampridine to treat Lambert-Eaton myasthenic syndrome. (Pubmed Central) -  Dec 15, 2020   
    The recommended dose is 80 mg a day, divided 3 or 4 times a day. Side effects are usually mild, and the most frequently reported are paresthesia.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Probing the Strength and Mechanism of Binding Between Amifampridine and Calf Thymus DNA. (Pubmed Central) -  Oct 26, 2020   
    Competitive fluorescence experiments using Acridine Orange (AO) and Hoechst 33258 (HO) probes also reveal that amifampridine binds to DNA via an intercalation mode of binding. Finally, the molecular docking studies also suggest that amifampridine tends to bind with the G-C rich region of DNA.