Companies similar to ACTION DUCHENNE
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  • www.synpharm.com
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Synergy Pharm specialises in medical and scientific writing. Qualified writers, proofreaders and translators for the writing of your medical documentation... Every member of our team is an experienced medical writer with a PhD, and all are native..
Relevance: 16.860477
  • cgtaresearchgroup.org
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Our goal is to bring gene therapies for rare Central Nervous System diseases into clinical trials and use as quickly and efficiently as possible. Our first project is focused on developing a gene therapy for Huntington's disease...
Relevance: 16.856363
  • www.clevelandcountywater.com
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Cleveland County Water will be closed on Monday, May 27, 2024 for the Memorial Day Holiday. The office will reopen on Tuesday, May 28, 2024 at 8:00 a.m...
Relevance: 16.84755
  • sobi-northamerica.com
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Sobi is a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families...
Relevance: 16.846302
  • tangoxstudy.com
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We believe collaboration is the only way forward to effectively tackle this rare and challenging disease entity. This is why our team founded the TANGO Consortium, an international network of patients, physicians, researchers, and industry partners..
Relevance: 16.813026
  • odylia.org
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Odylia was founded by Scott Dorfman, a successful entrepreneur and the father of two children with Usher syndrome, a rare genetic disease that affects vision and hearing, and Luk Vandenberghe, PhD, an innovator in the field of gene therapy. As a..
Relevance: 16.810408
  • www.raysopeningday.com
Wander Franco, Randy Arozarena, and Taj Bradley are gonna make Rays Opening Day 2024 the funnest one yet, and thanks to the availability of great tickets at affordable prices, you can see them do it, live at Tropicana Field in St. Petersburg, FL! The..
Relevance: 16.807693
  • www.emmausmedical.com
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Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for..
Relevance: 16.788233
  • www.biomarin-rareconnections.com
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BioMarin RareConnections TM helps you navigate the insurance process and identify assistance options you may be eligible for to start and continue on treatment... BioMarin RareConnections offers one-to-one financial navigation support for rare..
Relevance: 16.779099
  • pcdsupport.org.uk
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At PCD Support UK, we put our patient community first. We are a team of volunteers whose mission it is to support families and individuals affected by PCD. We also work closely with our clinical professionals, championing research to improve the..
Relevance: 16.761578
  • www.dayanandanshomeo.com
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We offer special consultation and treatment for varies disease like Celiac, Perthes, Hemophilia, Venereal, Psoriasis, Perianal Abscess etc.,... DR.DAYANANDAN'S HOMEOPATHY CLINIC A unique site for homeopathic treatment of rare diseases. We offer..
Relevance: 16.756483
  • www.arizonadiamondbacksopeningday.com
Love your Arizona Diamondbacks? Then you'll want to kick off the 2024 season with a big win over the Colorado Rockies! Tickets for Opening Day 2024 are on sale now, so you can be there live at Chase Field, cheering Ketel Marte and the boys to a big..
Relevance: 16.745518
  • www.lucanepharma.com
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Lucane Pharma's strategy for growth follows three main pathways: Successful development of own drugs, co-development of drugs and in- and out-licensing of medicinal products. Lucane Pharma is actively engaged in the development of 9 products, each of..
Relevance: 16.74193
  • biostherapeutics.com
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Bios Therapeutics is a NEXT-GENERATION Rare Diseases Pharmaceutical organization providing services and solutions for geographical expansion of small and mid-size companies and acting on their behalf on local or regional level... Bios Therapeutics..
Relevance: 16.741497
  • www.veraxresearch.com
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Verax specializes in non-THC cannabidiol research, rare disease product development, and laboratory proficiency testing. Our Redesign of Healthcare pricing and transparency models are unparalleled in the healthcare market... Verax has developed a..
Relevance: 16.741219
  • amrytpharma.com
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Amryt is a biopharmaceutical company dedicated to developing and delivering innovative new treatments to help improve the lives of patients with rare and debilitating diseases. Amryt comprises a profitable and growing commercial business with a..
Relevance: 16.729908
  • www.rd-rp.com
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Rare Disease Research Partners support research and access to treatment for people living with rare conditions... We provide patients with access to worldwide clinical trials by offering personalised logistic support to patients and their families..
Relevance: 16.718729
  • savarapharma.com
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Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead product candidate, molgramostim, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune..
Relevance: 16.701109
  • www.speragen.com
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Speragen was cofounded by a mother of two children who are affected with a rare disease, Succinic Semialdehyde Dehydrogenase (SSADH) deficiency. Speragen knows first-hand the challenges of navigating a life with a rare disease: from the daily..
Relevance: 16.699781
  • www.arpkdchf.org
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The ARPKD/CHF Alliance is a 501(c)(3) nonprofit, public charity organization that represents Autosomal Recessive Polycystic Kidney Disease (ARPKD) and Congenital Hepatic Fibrosis (CHF). We the patients and parents of affected children, and the..
Relevance: 16.698776
  • biomtherapeutics.com
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Biom Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies for brain disorders... Biom Therapeutics is founded by a team of business experts, scientists and clinicians with a focus on..
Relevance: 16.663176
  • mass-team.com
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The MASS Team are a specialist rare disease marketing and access consultancy generating actionable marketing and access strategies that are underpinned by patient, KOL and payer insight, and our specialist pharma industry knowledge and experience.....
Relevance: 16.662464
  • floricatherapeutics.com
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We have created in a dish a novel brain cell component that can be used to encapsulate drugs, specifically biologics and gene therapy products. Our invention makes these products amenable for delivery across the blood brain barrier. Our beachhead..
Relevance: 16.631954
  • alzheimergenetics.org
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Our aim is to identify rare high-impact variants that play a role in Alzheimer's disease and other neurodegenerative diseases, in order to increase our understanding of disease etiology and improve clinical diagnosis/prognosis... For this purpose,..
Relevance: 16.626423
  • alsfund.ru
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The Live Now Foundation provides comprehensive support to people with ALS (amyotrophic lateral sclerosis) throughout Russia. ALS, better known as Stephen Hawking's disease, is a rare incurable disease affecting motor neurons that transmit the..
Relevance: 16.619076
  • rare-diseases.ca
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Our Emerging Team in Rare Diseases aims to undertake collaborative translational research with the objective of helping to inform the development of drug coverage decision-making models relating to government funding of drug therapy for rare..
Relevance: 16.564701
  • rosebudbiosci.com
Rosebud Biosciences is an early stage startup that develops therapies for rare genetic diseases in children. We do this by modeling human development with organoids that have the same gene mutation as the child, and screen drugs in these models to..
Relevance: 16.564579
  • kamaripharma.com
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Our management team is comprised of individuals with vast experience in drug discovery and pharmaceutical development in the biotech industry, with focus on skin disorders and rare disease. We are supported by clinical and scientific advisors who are..
Relevance: 16.556099
  • www.stategics.com
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STATegics, Inc. is focused on the discovery and development of breakthrough new treatments for life-threatening rare neurodegenerative diseases. By targeting the tissue-protective cytokine receptor, STATegics has identified novel small molecules to..
Relevance: 16.548986
  • wambachcolelab.wustl.edu
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Our lab is interested in understanding the genetic causes of rare diseases and birth defects among infants and children. Our research goals are to use genomic sequencing technologies including whole exome and genome sequencing, RNASeq and gene..
Relevance: 16.530186