Confirmed Supporters

Confirmed Exhibitors

	Alexion Pharma GmbH \| Booth #9-10 Alexion is a global biopharmaceutical company focused on developing and delivering lifetransforming therapies for patients with devastating and rare disorders. Alexion has two complement inhibitors and a metabolic franchise with two enzyme replacement therapies. Alexion focuses its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. (alexion.com)
	Audentes Therapeutics \| Booth #21 (audentestx.com)
	AveXis, Inc. \| Booth #23 AveXis is a gene therapy company focused on developing novel treatments and ensuring access for patients and families affected by rare and life-threatening neurological genetic diseases. Our goal is to utilise cutting-edge science to deliver a functional copy of a lost or defective gene, using a single-dose delivery method. (avexis.com)
	Biogen \| Booths #5-7 & #6-8 At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapiesfor people living with serious neurological and neurodegenerative diseases. (biogen.com)
	DEYMED Diagnostic \| Booth #4 The company was founded in 1997 and its first product was developing a 32 channel EEG device which was completed and sold in 1998. In the following years additional neurology products have been developed including EMG, PSG and TMS devices. (deymed.com)
	EURO NMD (European Reference Network for Neuromuscular Diseases) (ern-euro-nmd.eu)
	ICNMD 2022 (icnmd.org)
	IOS Press (iospress.nl)
	iThera Medical \| Booth #13 iThera Medical’s biomedical imaging technology MSOT (multispectral optoacoustic tomography) allows for in-vivo real-time identification of spectral signatures from various tissue chromophores, including hemoglobin, lipids, collagen, and water. The technology is used in a wide range of preclinical and clinical applications, including cancer, inflammatory and vascular diseases. Recent research suggests that it can also be used to study neuromuscular disease progression by quantifying collagen content in muscle tissue. (ithera-medical.com)
	LFB \| Booth #27 LFB develops, manufactures and markets therapeutic proteins for patients with serious and often rare diseases. LFB is among the leading European biopharmaceutical companies providing mainly hospital-based healthcare professionals with plasma-derived or recombinant medicinal products in three major therapeutic areas: immunology, haemostasis, and intensive care. (groupe-lfb.com)
	LUPIN NEUROSCIENCES \| Booth #11 Launching NaMuscla® in Europe as the first licensed antimyotonic globally established Lupin Neurosciences as a new key stakeholder in the neuromuscular field. This specialty pharma division of Lupin aims to build on the launch of the company’s first Orphan Drug by developing a global portfolio of science-based solutions in neuromuscular diseases and movement disorders. (lupinneurosciences.com)
	Pharnext \| Booth #14 Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for neurodegenerative diseases. The company developed a new drug discovery paradigm that identifies and develops synergic combinations of drugs. PXT3003 completed an international Phase 3 trial with positive topline results for CMT1A and PXT864 generated encouraging Phase 2 results in Alzheimer’s disease. (pharnext.com)
	PTC Therapeutics \| Booth #24 PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. (ptcbio.com)
	ReveraGen BioPharma \| Booth #3 ReveraGen BioPharma is developing vamorolone, an oral anti-inflammatory drug and mineralocorticoid antagonist, for Duchenne muscular dystrophy. In open label trials, vamorolone led to improvements in gross motor outcomes over 18 months of treatment, with loss of safety concerns associated with chronic corticosteroids. The pivotal trial is underway in DMD. (reveragen.com)
	Roche \| Booth #15-18 Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. (roche.com)
	Sanofi Genzyme \| Booth #20-22 Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families. (sanofigenzyme.com)
	Santhera \| Booth #19 Santhera Pharmaceuticals (SIX: SANN) focuses on developing innovative medicines for rare neuromuscular and pulmonary diseases. In Europe, Puldysa® (idebenone) is under regulatory review by the EMA for Duchenne muscular dystrophy. The pipeline includes rights to dissociative steroid vamorolone for DMD, POL6014 for cystic fibrosis and omigapil for congenital muscular dystrophy. (santhera.com)
	Sarepta \| Booth #2 Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in Limb-girdle muscular dystrophy (LGMD), Charcot-Marie-Tooth (CMT) MPS IIIA, Pompe and other CNSrelated disorders, totaling over 20 therapies in various stages of development. (sarepta.com)
	Takeda \| Booth #12 Takeda is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology, Neuroscience and Rare Diseases and targeted R&D-investments in Plasma-Derived-Therapies and Vaccines. (takeda.com)
	UCB Pharma Ltd. \| Booth #30 UCB is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. Follow us on Twitter: @UCB_news. (ucb.com)
	University of Florida Shands Hospital (ufhealth.org)

	Alexion Pharma GmbH \| Booth #9-10 Alexion is a global biopharmaceutical company focused on developing and delivering lifetransforming therapies for patients with devastating and rare disorders. Alexion has two complement inhibitors and a metabolic franchise with two enzyme replacement therapies. Alexion focuses its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. (alexion.com)
	Audentes Therapeutics \| Booth #21 (audentestx.com)
	AveXis, Inc. \| Booth #23 AveXis is a gene therapy company focused on developing novel treatments and ensuring access for patients and families affected by rare and life-threatening neurological genetic diseases. Our goal is to utilise cutting-edge science to deliver a functional copy of a lost or defective gene, using a single-dose delivery method. (avexis.com)
	Biogen \| Booths #5-7 & #6-8 At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapiesfor people living with serious neurological and neurodegenerative diseases. (biogen.com)
	DEYMED Diagnostic \| Booth #4 The company was founded in 1997 and its first product was developing a 32 channel EEG device which was completed and sold in 1998. In the following years additional neurology products have been developed including EMG, PSG and TMS devices. (deymed.com)
	EURO NMD (European Reference Network for Neuromuscular Diseases) (ern-euro-nmd.eu)
	ICNMD 2022 (icnmd.org)
	IOS Press (iospress.nl)
	iThera Medical \| Booth #13 iThera Medical’s biomedical imaging technology MSOT (multispectral optoacoustic tomography) allows for in-vivo real-time identification of spectral signatures from various tissue chromophores, including hemoglobin, lipids, collagen, and water. The technology is used in a wide range of preclinical and clinical applications, including cancer, inflammatory and vascular diseases. Recent research suggests that it can also be used to study neuromuscular disease progression by quantifying collagen content in muscle tissue. (ithera-medical.com)
	LFB \| Booth #27 LFB develops, manufactures and markets therapeutic proteins for patients with serious and often rare diseases. LFB is among the leading European biopharmaceutical companies providing mainly hospital-based healthcare professionals with plasma-derived or recombinant medicinal products in three major therapeutic areas: immunology, haemostasis, and intensive care. (groupe-lfb.com)
	LUPIN NEUROSCIENCES \| Booth #11 Launching NaMuscla® in Europe as the first licensed antimyotonic globally established Lupin Neurosciences as a new key stakeholder in the neuromuscular field. This specialty pharma division of Lupin aims to build on the launch of the company’s first Orphan Drug by developing a global portfolio of science-based solutions in neuromuscular diseases and movement disorders. (lupinneurosciences.com)
	Pharnext \| Booth #14 Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for neurodegenerative diseases. The company developed a new drug discovery paradigm that identifies and develops synergic combinations of drugs. PXT3003 completed an international Phase 3 trial with positive topline results for CMT1A and PXT864 generated encouraging Phase 2 results in Alzheimer’s disease. (pharnext.com)
	PTC Therapeutics \| Booth #24 PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. (ptcbio.com)
	ReveraGen BioPharma \| Booth #3 ReveraGen BioPharma is developing vamorolone, an oral anti-inflammatory drug and mineralocorticoid antagonist, for Duchenne muscular dystrophy. In open label trials, vamorolone led to improvements in gross motor outcomes over 18 months of treatment, with loss of safety concerns associated with chronic corticosteroids. The pivotal trial is underway in DMD. (reveragen.com)
	Roche \| Booth #15-18 Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. (roche.com)
	Sanofi Genzyme \| Booth #20-22 Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families. (sanofigenzyme.com)
	Santhera \| Booth #19 Santhera Pharmaceuticals (SIX: SANN) focuses on developing innovative medicines for rare neuromuscular and pulmonary diseases. In Europe, Puldysa® (idebenone) is under regulatory review by the EMA for Duchenne muscular dystrophy. The pipeline includes rights to dissociative steroid vamorolone for DMD, POL6014 for cystic fibrosis and omigapil for congenital muscular dystrophy. (santhera.com)
	Sarepta \| Booth #2 Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in Limb-girdle muscular dystrophy (LGMD), Charcot-Marie-Tooth (CMT) MPS IIIA, Pompe and other CNSrelated disorders, totaling over 20 therapies in various stages of development. (sarepta.com)
	Takeda \| Booth #12 Takeda is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology, Neuroscience and Rare Diseases and targeted R&D-investments in Plasma-Derived-Therapies and Vaccines. (takeda.com)
	UCB Pharma Ltd. \| Booth #30 UCB is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. Follow us on Twitter: @UCB_news. (ucb.com)
	University of Florida Shands Hospital (ufhealth.org)

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