李宗㟨精品72集,黄图能看见奶头

Investors and Media

Scientific publications

Idebenone (Puldysa) in Duchenne muscular dystrophy (DMD)

Papers

Servais L, Straathof CSM, Schara U, et al. (2020). Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy. Neuromuscular Disorders 30:5-16.

Buyse GM, Rummey C, Meier T, et al. (2018). Home-based monitoring of pulmonary function in patients with Duchenne muscular dystrophy. Journal of Neuromuscular Diseases 5:419–430.

Mayer OH, Leinonen M, Rummey C, et al. (2017). Efficacy of idebenone to preserve respiratory function above clinically meaningful thresholds for Forced Vital Capacity (FVC) in patients with Duchenne muscular dystrophy. Journal of Neuromuscular Diseases李宗㟨精品72集,黄图能看见奶头 4: 189–198.

Meier T, Rummey C, Leinonen M, et al. (2017). Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy. Neuromuscular Disorders 27: 307–314.

Mayer OH, Henricson EK, McDonald CM and Buyse GM (2017). Advances in pulmonary care in Duchenne muscular dystrophy. US Neurology 13:35–41.

Buyse GM, Voit T, Schara U, et al. (2017). Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy. Pediatric Pulmonology李宗㟨精品72集,黄图能看见奶头 52:508–515.

McDonald CM, Meier T, Voit T, et al. (2016). Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy. Neuromuscular Disorders 26: 473–480.

Buyse GM, Gueven N, McDonald CM (2015). Idebenone as a novel therapeutic approach for Duchenne muscular dystrophy. European Neurological Review李宗㟨精品72集,黄图能看见奶头 10(2):189–94.

Buyse GM, Voit T, Schara U, et al. (2015). Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Lancet 李宗㟨精品72集,黄图能看见奶头385:1748–1757.

Mayer OH, Finkel RS, Rummey C, et al. (2015). Characterization of pulmonary function in Duchenne muscular dystrophy. Pediatric Pulmonology李宗㟨精品72集,黄图能看见奶头 50:487–494.

Buyse GM, Goemans N, van den Hauwe M and Thomas Meier (2013). Effects of glucocorticoids and idebenone on respiratory function in patients with Duchenne muscular dystrophy. Pediatric Pulmonology李宗㟨精品72集,黄图能看见奶头 48: 912–20.

Buyse GM, Goemans N, van den Hauwe M, et al. (2011). Idebenone as a novel therapeutic approach for Duchenne muscular dystrophy: Results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscular Disorders李宗㟨精品72集,黄图能看见奶头 21: 396–405.

Buyse GM, Van der Mieren G, Erb M (2009). Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance. European Heart J 30: 116-24.

Conferences

World Muscle Society (WMS) Annual Congress
Oct 1-5, 2019 (Copenhagen, Denmark)
Poster #P261: Buyse et al.: Consistent long-term effect of idebenone in reducing respiratory function decline in advanced patients with Duchenne muscular dystrophy (DMD)
Poster #P264李宗㟨精品72集,黄图能看见奶头: Servais et al.: SYROS study -- long-term reduction in rate of respiratory function decline in patients with Duchenne muscular dystrophy (DMD) treated with idebenone

European Respiratory Society (ERS) International Congress
Sep 28 - Oct 2, 2019 (Madrid, Spain)
Poster #5330: Mayer et al.: Evaluating the effect of long-term idebenone treatment on respiratory morbidity in patients with Duchenne muscular dystrophy (DMD)
Poster #5331: Mayer et al.: Long term efficacy: Idebenone reduces the rate of both inspiratory and expiratory functional loss in Duchenne muscular dystrophy (DMD)

13th European Pediatric Neurology Society (EPNS) Congress
Sep 18-21, 2019 (Athens, Greece)
Poster #138: Long term analysis of the rate of respiratory function decline in patients with Duchenne muscular dystrophy (DMD) in a real-world setting: The SYROS study
Poster #140: Consistent long-term effect of idebenone on the rate of respiratory function decline in advanced patients with Duchenne muscular dystrophy (DMD)

2019 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference
Apr 13-17, 2019 (Orlando, USA)
Poster #7: SYROS study – long-term reduction in rate of respiratory function decline in patients with Duchenne Muscular Dystrophy (DMD) treated with idebenone
Poster #9: Consistent long-term effect of idebenone in reducing respiratory function decline in advanced patients with Duchenne Muscular Dystrophy (DMD)
Poster #11: Crossing thresholds and changing rates of respiratory function decline are predictive of clinical outcomes in Duchenne Muscular Dystrophy (DMD)

Vamorolone in Duchenne muscular dystrophy (DMD)

Papers

Hoffman EP, Schwartz BD, Mengle-Gaw LJ, et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93: e1312-e1323.

Mavroudis PD, van den Anker J, Conklin LS, et al. (2019). Population pharmacokinetics of vamorolone (VBP15) in healthy men and boys with Duchenne muscular dystrophy. J Clin Pharmacol. 59 :979-988.

Heier CR, Yu Q, Fiorillo AA, et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance vol 2 | no 1 | e201800186

Hoffman EP, Riddle V, Siegler MA, et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids李宗㟨精品72集,黄图能看见奶头 134: 43–52.

Conklin LS, Damsker JM, Hoffman EP, et al. (2018). Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacological Research 136: 140–150.

Streetama SC, Chandra G, van der Meulen JH, et al. (2018). Membrane stabilization by modified steroid offers a potential therapy for muscular dystrophy due to dysferlin deficit. Molecular Therapy 26: 2231-2242.

Heier CR, Damsker JM, You Q, et al. (2013). VBP15, a novel anti‐inflammatory and membrane‐stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med李宗㟨精品72集,黄图能看见奶头. 5: 1569–1585

Reeves EKM, Hoffman EP, Nagaraju K, et al. (2013). VBP15: Preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem. 21: 2241–2249.

Lonodelestat (POL6014)

Papers

Barth P. et al (2019). Single dose escalation studies with inhaled POL6014, a potent novel selective reversible inhibitor of human neutrophil elastase, in healthy volunteers and subjects with cystic fibrosis. J Cyst Fibros. Sep 6. pii: S1569-1993(19)30878-1. doi: 10.1016/j.jcf.2019.08.020.

Conferences

2018 American Thoraic Society International Conference
May 21, 2018 (San Diego, USA)
Poster #A2988/P878 Effect of POL6014, a potent and selective inhaled neutrophil elastase inhibitor, in a rat model of lung neutrophil activation

2009 American Thoraic Society International Conference
May 15-20, 2009 (San Diego, USA)
Poster A novel Protein Epitope Mimetic (PEM) neutrophil elastase (NE) inhibitor, POL6014, inhibits human NE-induced acute lung injury in mice.

Congenital muscular dystrophy (CMD)

Papers

Meinen S, Lin S, Thurnherr R, et al. (2011). Apoptosis inhibitors and mini-agrin have additive benefits in congenital muscular dystrophy mice. EMBO Molecular Medicine 3:465-479.

Erb M, Meinen S, Barzaghi P, et al. (2009). Omigapil ameliorates the pathology of muscle dystrophy caused by Laminin-alpha2 deficiency. J Pharmacology Experimental Therapeutics 331:787–795.

Idebenone (Raxone) in Leber's hereditary optic neuropathy (LHON)

Papers

Klopstock T, Metz G, Yu-Wai-Man P et al. (2013). Persistence of the treatment effect of idebenone in Leber’s hereditary optic neuropathy. Brain (Pt 2):e230.

Rudolph G, Dimitriadis K, Büchner B, et al. (2013). Effects of idebenone on color vision in patients with Leber Hereditary Optic Neuropathy. Journal of Neuro-Ophthalmology 33: 30-36.

Klopstock T, Yu-Wai-Man P, Dimitriadis K, et al. (2011). A randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy. Brain 134: 2677–2686.

Mascialino B, Leinonen M, Meier T (2012). Meta-analysis of the prevalence of Leber’s hereditary optic neuropathy mtDNA mutations in Europe. European Journal Ophthalmology 22: 461-465.

Conferences

EUNOS - 14th Meeting of European Neuro-Ophthalmological Society
Jun 16-19, 2019 (Porto, Portugal)
Poster P052: Long-term (over 24 months) treatment with idebenone may continue to improve visual function response in patients with Leber’s Hereditary Optic Neuropathy (LHON)
Poster P053: Responder analysis of chronic Leber’s hereditary optic neuropathy (LHON) patients to idebenone in a placebo controlled, randomized clinical trial (RHODOS)
Poster P054: Pediatric Leber’s Hereditary Optic Neuropathy (LHON): Real-world efficacy results following long term idebenone treatment
Poster P055: Evaluating long-term real-world data (RWD) from patients with Leber’s hereditary optic neuropathy (LHON) in light of the recommendations of the International Consensus statement
Poster PAG: A Patient Centric Activity in the Development and Lifecycle of Medicines

NANOS - 45th Annual Meeting of the North-American Neuro-Ophthalmology Society
Mar 16-21, 2019 (Las Vegas, USA)
Scientific poster presentations.