Logo Medical XPRT
Companies
Products
Services
Software
Training
Applications
Sign in
List your business

Advertising options
  1. Home
  2. Companies
  3. rare disease
Show results for
Products
Services
Software
Training
Applications

Companies

News
Articles
Downloads
Videos

Refine by
Locations

  • USA
  • Canada
  • Europe

  • Africa
  • Asia & Middle East
  • Australasia
  • Latin America
  • North America
Business Types

  • Manufacturer
  • Technology
  • Service provider
  • Software vendor
  • Training provider
  • Distributor
  • Consulting firm
  • Nonprofit organization (NPO)
Industries served

  • Medical / Health Care
  • Chemical & Pharmaceuticals
  • Environmental
  • Banking & Finance / Insurance / Legal
  • Aerospace & Air Transport
  • Retail
  • Waste and Recycling
  • Monitoring and Testing
  • Health and Safety
  • Air and Climate
  • Soil and Groundwater
  • Food and Beverage
  • Energy
  • Construction & Construction Materials
Employees

  • 101-1000
  • Over 1000

Rare Disease Suppliers & Manufacturers

94 companies found

Azafaros

Azafaros

based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Oral Treatment for Lysosomal Storage Disorders

Oral Treatment for Lysosomal Storage Disorders

Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique ...
CONTACT SUPPLIER

Orgenesis Inc.

Orgenesis Inc.

based inGermantown, MARYLAND (USA)
Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues like cancer, genetic disorders, immunodeficiencies, rare diseases, and more. CGTs have shown potential to replace damaged cells with ...
CONTACT SUPPLIER

Rentschler Biopharma SE

Rentschler Biopharma SE

based inLaupheim, GERMANY
We are a leading global Contract Development and Manufacturing Organization (CDMO), focused exclusively on client projects. From our headquarters in Laupheim, Germany, and our site in Milford, MA, USA, we offer end-to-end solutions including ...
CONTACT SUPPLIER

MitoImmune Therapeutics Inc.

MitoImmune Therapeutics Inc.

based inGangnam-gu, SOUTH KOREA
MitoImmune Therapeutics Inc. is a drug discovery and development biotech company that harnesses the power of mitochondria and its proprietary technology targeting mitochondria as the source for developing new potential breakthrough therapeutics. Our ...
CONTACT SUPPLIER

CycloLab Cyclodextrin Research and Development Laboratory Ltd.

CycloLab Cyclodextrin Research and Development Laboratory Ltd.

based inBudapest, HUNGARY
CycloLab is a worldwide recognized and all-around cyclodextrin research and development company, having the longest tradition and broadest circle of customers. Since 1972, a staff of internationally acknowledged experts have been working in the ...
CONTACT SUPPLIER

Evox Therapeutics Limited

Evox Therapeutics Limited

based inOxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Evox - Model Type C - Lysosomal Storage Disorder

Evox - Model Type C - Lysosomal Storage Disorder

Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
CONTACT SUPPLIER

Virpax Pharmaceuticals

Virpax Pharmaceuticals

based inBerwyn, PENNSYLVANIA (USA)
Virpax Pharmaceuticals specializes in developing novel and proprietary drug-delivery systems across various pain and CNS disorder indications to enhance compliance and to optimize the efficacy of each product candidate. Our transforming ...
CONTACT SUPPLIER

Amicus Therapeutics, Inc.

Amicus Therapeutics, Inc.

based inPhiladelphia, PENNSYLVANIA (USA)
We are a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and ...
CONTACT SUPPLIER

Applied Pharma Research s.a.

Applied Pharma Research s.a.

based inBalerna, SWITZERLAND
APR Applied Pharma Research s.a. (APR) is a Swiss Pharma company dedicated to providing patients suffering from rare diseases with innovative treatments that make a real difference in their life. Our focus as a Swiss global health care company is ...
CONTACT SUPPLIER

Woolsey Pharmaceuticals, Inc.

Woolsey Pharmaceuticals, Inc.

based inNew York City, NEW YORK (USA)
Woolsey Pharmaceuticals is focused on the treatment of rare, neurodegenerative diseases. Many neurodegenerative diseases – including Alzheimer’s, vascular dementia, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Huntington’s disease, ...
CONTACT SUPPLIER

Strand Life Sciences Pvt. Ltd.

Strand Life Sciences Pvt. Ltd.

based inBangalore, INDIA
Strand Life Sciences is a genomics-based research and diagnostics company with a mission to improve global health by decoding the genetic information contained in an individual’s genome. Strand Life Sciences partnership programs enable large-scale ...
SmartLab Services

SmartLab Services

Strand has extensive experience in validating NGS-based panels under CAP- and CLIA-settings: be it germline panels, somatic tumor panels, or liquid biopsy panels, and be it small panels with tens of genes, to large panels with thousands of ...
CONTACT SUPPLIER

Infant Bacterial Therapeutics AB

Infant Bacterial Therapeutics AB

based inStockholm, SWEDEN
IBT is a pharmaceutical company with its registered office in Stockholm with a vision to develop drugs influencing the human infant microbiome, and thereby prevent or treat rare diseases affecting premature infants. IBT is currently developing its ...
Model IBP-9414 - Preventing Necrotizing Enterocolitis

Model IBP-9414 - Preventing Necrotizing Enterocolitis

IBP-9414 for the prevention of necrotizing enterocolitis (NEC) contains the active substance Lactobacillus reuteri, which is a co-evolved human bacterial strain naturally present in breast milk. Lactobacillus reuteri is a live bacteria known to be ...
CONTACT SUPPLIER

Andelyn Biosciences

Andelyn Biosciences

based inColumbus, OHIO (USA)
Andelyn Biosciences is a biopharmaceutical CDMO Pioneering Solutions That Turn Hope into Reality™. With 20+ years of experience manufacturing viral vectors, Andelyn’s scientific expertise for development and characterization has led to GMP material ...
Clinical Manufacturing Services

Clinical Manufacturing Services

Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV and Lentiviral adherent and suspension systems along with plasmid manufacturing. We support our clients throughout every ...
CONTACT SUPPLIER

Harmony Biosciences

Harmony Biosciences

based inPlymouth Meeting, PENNSYLVANIA (USA)
Harmony Biosciences is a commercial-stage pharmaceutical company dedicated to developing and delivering innovative therapies for people living with rare neurological diseases, especially sleep/wake, neurobehavioral, and rare epilepsy disorders. The ...
CONTACT SUPPLIER

Oncodesign

Oncodesign

based inDijon, FRANCE
Oncodesign is a biopharmaceutical company that offers you powerful technologies and tools to guide you through the development of your drug, from medicinal chemistry to drug candidate. Our therapeutic areas are expanding. In addition to oncology, ...
Oncodesign - Model Chi-Mice - Humanized Technology

Oncodesign - Model Chi-Mice - Humanized Technology

This is the best tool you can use to evaluate new therapeutic approaches in conditions that are as close as possible to a clinical situation: ...
CONTACT SUPPLIER

Canè S.p.A.

Canè S.p.A.

based inRivoli (TO), TURKEY
Since 1978, manufacturer of CRONO pumps, portable infusion systems for the treatment of several diseases. All this has led to the creation of a series of drug-delivery pumps dedicated to the treatment of various pathologies. Canè microinfusers ...
CONTACT SUPPLIER

KORU Medical Systems

KORU Medical Systems

based inMahwah, NEW JERSEY (USA)
KORU is, first and foremost, a patient-centric organization that will go the extra mile for our customers, providers, and partners who depend on our devices. We are a world-class manufacturer of subcutaneous infusion devices that deliver life-saving ...
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Chronic inflammatory demyelinating polyneuropathy, or CIDP, is a rare disorder characterized by gradually increasing sensory loss and weakness associated with loss of reflexes. This is caused by an autoimmune process in which the body’s own ...
CONTACT SUPPLIER

Forge Biologics, Inc.

Forge Biologics, Inc.

based inGrove City, OHIO (USA)
Forge is developing innovative gene therapies that aim to help patients suffering from devastating rare diseases. Forge is building capacity to directly address the growing demand for gene therapy manufacturing. Forge is currently building the ...
CONTACT SUPPLIER

Precision NanoSystems

Precision NanoSystems

based inVancouver, BRITISH COLUMBIA (CANADA)
Precision NanoSystems is a global leader in technologies, solutions and services for the development of non-viral delivery of genomic medicines, including mRNA vaccines and therapeutics. We work with the world’s leading drug developers to understand ...
CONTACT SUPPLIER

diaserve Laboratories GmbH

diaserve Laboratories GmbH

based inIffeldorf, GERMANY
diaserve laboratories is one of Europe’s leading suppliers of customized IVD calibrators and controls based on human sera. In constant cooperation with renowned institutes, we use the most up-to-date research findings to continuously further develop ...
Diaserve - Bulk Disease State Plasma Units

Diaserve - Bulk Disease State Plasma Units

Human products are frozen liquid materials based on voluntary blood donations. Materials are intended to support the industries to manufacture their own controls and calibrators. All listed products are selected and processed according to customer's ...
CONTACT SUPPLIER
  • Previous
  • Next
Need help finding the right suppliers?Try XPRT Sourcing. Let the XPRTs do the work for you
Back to top
About Medical XPRT

Medical XPRT is a global marketplace with solutions and suppliers for the medical sector, with product catalogs, articles, industry events, publications & more.

Channels

Solutions

  • Medical Equipment & Supplies
  • Medical Software
  • Medical Training Courses
  • Medical Industry Services
  • Medical Product Applications

Latest

  • Medical Industry News
  • Medical Industry Events

Publications

  • Medical Industry Articles & Case Studies
  • Medical Industry Books and Publications
  • Medical Industry Journals and Magazines
  • Medical Industry Downloads
  • Medical Industry Videos

Companies

  • Medical Industry Suppliers & Manufacturers

  • FAQs
  • Company sitemap
  • Category sitemap
Medical XPRT is part of XPRT Media All Rights Reserved.
Terms
Privacy
  • Medical XPRT RSS List
Our sites:
Environmental XPRT
Agriculture XPRT
Energy XPRT
XPRT