Companies similar to ACERAGEN
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  • sixerapharma.com
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Sixera Pharma is a small biopharmaceutical company and we want to develop our lead compound SXR1096 in an attractive skin cream as the first treatment available for Netherton syndrome. An ultra rare disease with a high unmet medical need... Sixera..
Relevance: 31.30589
  • travere.com
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Travere Therapeutics is a biopharmaceutical company whose mission is to identify, develop and deliver life-changing therapies to people living with rare disease... SAN DIEGO, Feb. 28, 2022 (GLOBE NEWSWIRE) - Travere Therapeutics, Inc. (NASDAQ: TVTX)..
Relevance: 30.0
  • therismedical.com
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Theris Medical is a clinical-stage biopharmaceutical company creating solutions for wound care. Our mission is to help an underserved patient group which suffers from wound complications from a rare disease - sickle cell anemia... Theris Medical is..
Relevance: 28.416752
  • 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: 28.224371
  • eusapharma.com
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Eusa Pharma is part of Recordati Rare Diseases, the business unit dedicated to rare disease in the Recordati group. Recordati has a total staff of more than 4,300, dedicated to the research, development, manufacturing and marketing of..
Relevance: 27.826788
  • www.ptcregistration.com
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PTC is a science-led, patient-centered biopharmaceutical company focused on discovering, developing and commercializing medicine for rare-disease patients... PTC Therapeutics is committed to providing valuable, expert-led education for healthcare..
Relevance: 27.191542
  • www.numericbiotech.com
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Numeric Biotech is a biopharmaceutical company fully dedicated to cure age-related diseases. In particular where chronic (neuro) inflammation is a root cause of the disease or is aggravating the severity of the disease... Numeric biotech is a..
Relevance: 27.012947
  • www.pharming.com
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Pharming is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies..
Relevance: 25.764196
  • 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: 25.727947
  • 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: 25.200006
  • www.recordatirarediseases.com
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Recordati Rare Diseases Inc. (RRD) is a biopharmaceutical company committed to providing therapies to the underserved and often overlooked rare disease communities of the United States. Recordati Rare Diseases is part of the rare diseases business..
Relevance: 23.980938
  • www.qolmed.com
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QOL Medical is a specialty biopharmaceutical company dedicated to improving each patient's life. We focus on solving complex, rare disease challenges with elegant solutions and want to change the world, one patient at a time... QOL Medical was..
Relevance: 23.699469
  • www.aceragen.com
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Aceragen is a rare disease biopharmaceutical company. We address rare, orphan diseases that typically have no available treatment, and strive to develop novel therapies which can have a positive impact on the lives of patients and families. As such,..
Relevance: 23.276756
  • www.neurolixis.com
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Neurolixis is an early-stage biopharmaceutical company focused on the discovery and development of novel drugs for the treatment of human central nervous system disorders including Parkinson's disease, rare autism spectrum disorders, depression and..
Relevance: 23.033169
  • www.tonixpharma.com
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Tonix is a biopharmaceutical company focused on developing novel therapeutics to prevent and treat central nervous system disorders, immunology conditions, infectious diseases, and rare diseases... Tonix is dedicated to finding cures for difficult..
Relevance: 22.917818
  • alexion.com
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Alexion has operations in the United Arab Emirates to serve patients throughout the Middle East and North Africa (MENA) region. Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for..
Relevance: 22.53396
  • www.engagehealth.com
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Engage Health is a health research and market development firm who has helped companies in the rare disease space for over 25 years... Engage Health's team provides comprehensive services to further development of rare and specialty disease..
Relevance: 21.901588
  • scantox.com
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Scantox is your guarantee for solid timelines, on-time and on-budget deliveries, the highest quality scientific data, and a close partnership where you are in contact with a dedicated scientist from start to finish of your study... Scantox is..
Relevance: 21.87731
  • kitherbiotech.com
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Kither Biotech is a biopharmaceutical company developing signal transduction modulators for the treatment of rare respiratory diseases...
Relevance: 21.784506
  • www.strykagen.com
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Located within the beautiful Truckee Meadows in the Reno-Tahoe area, Strykagen is a biopharmaceutical company devoted to the development of transforming therapeutics and diagnostics for life-threatening rare muscle diseases. There is currently an..
Relevance: 21.624088
  • www.rezolutebio.com
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Rezolute is a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (HI). Our antibody therapy, RZ358 (ersodetug), is designed to treat all forms of HI and has shown..
Relevance: 21.564833
  • www.khondrion.com
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Khondrion is a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease...
Relevance: 21.39469
  • rarediseasesday.wustl.edu
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According to the National Institutes of Health (NIH), a disease is rare if it affects less than 200,000 people in the United States. Over 25 million Americans live with a rare disease-and nearly half of these patients are children. For the over 7,000..
Relevance: 21.372194
  • www.amberophthalmics.com
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Amber Ophthalmics is a clinical stage biopharmaceutical company formed in 2020, focused on driving novel therapeutics for rare anterior segment ophthalmic diseases...
Relevance: 21.241453
  • www.swii.ch
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Swii.ch health is a rare disease patient insight agency and strategy consultancy. We help our healthcare clients make better informed decisions based on a deep understanding of human behaviour... In addition to our core team, we work with a wider..
Relevance: 21.207308
  • cleavetherapeutics.com
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Cleave Therapeutics is a clinical stage biopharmaceutical company focused on valosin-containing protein (VCP)/p97 as a novel target in cellular stress pathways essential to cancer cell growth and survival such as the DNA damage response (DDR) and the..
Relevance: 21.188599
  • www.soligenix.com
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Soligenix is a late-stage biopharmaceutical company committed to developing and commercializing products to treat rare diseases where there is an unmet medical need...
Relevance: 21.100462
  • finding-rare.com
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OUr Team Our team's combined 40+ years in the rare disease marketplace and personal experience with the rare disease diagnostic odyssey led us to believe that there had to be a better way. We knew the data to accelerate rare disease diagnoses and..
Relevance: 21.082905
  • www.galleonbio.com
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GalleonBio is a mid-stage biopharmaceutical company focusing on the development of novel topical therapies for the management of inflammatory dermatological, neuropathic pain and other conditions. Based on multiple years of research and scientific..
Relevance: 21.023745
  • www.alixalderman.com
Alix Alderman Kentfield is a longstanding presence in the biopharmaceutical sphere who has held leadership responsibilities with InterMune, Amylin, Abbott/AbbVie, Ultragenyx, BridgeBio, PellePharm, Neumora, and the Board of Directors at TOPRA. She..
Relevance: 20.986292