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Rare Disease Suppliers & Manufacturers

94 companies found

Suvoda

Suvoda

based inConshohocken, PENNSYLVANIA (USA)
Suvoda is a global clinical trial technology company dedicated to supporting complex studies for life-sustaining therapies.We shape digital experiences that empower you to take command over mission-critical and time-sensitive patient interactions in ...
Model IRT - Advanced Patient Randomization and Trial Supply Management System

Model IRT - Advanced Patient Randomization and Trial Supply Management System

Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...
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

Regeneron Pharmaceuticals Inc.

Regeneron Pharmaceuticals Inc.

based inTarrytown, NEW YORK (USA)
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently ...
CONTACT SUPPLIER

Acorda Therapeutics, Inc.

Acorda Therapeutics, Inc.

based inPearl River, NEW YORK (USA)
Acorda Therapeutics is a publicly traded (NASDAQ: ACOR) biotechnology company whose mission is to develop therapies that restore neurological function and improve the lives of people with neurological disorders. We started our company in 1995 as a ...
CONTACT SUPPLIER

Population Bio, Inc. a Precision Medicine Company

Population Bio, Inc. a Precision Medicine Company

based inMelville, NEW YORK (USA)
Population Bio, Inc. is a privately held precision medicine company that is empowering pharmaceutical companies to develop targeted therapies and companion diagnostics faster and more cost effectively. We are accelerating the delivery of precision ...
CONTACT SUPPLIER

Lumos Pharma

Lumos Pharma

based inAustin, TEXAS (USA)
Lumos Pharma is a clinical-stage biopharmaceutical company whose mission is to develop new therapies for people with rare diseases, prioritizing its focus where the medical need is high, and the pathophysiology is clear. We are committed to this ...
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

Palvella Therapeutics.

Palvella Therapeutics.

based inWayne, PENNSYLVANIA (USA)
Palvella Therapeutics was founded to serve individuals suffering from rare diseases by developing safe and efficacious therapies that will dramatically enhance their quality of life. We focus on developing therapies that specifically target the root ...
Palvella - Model QTORIN - Gorlin Syndrome

Palvella - Model QTORIN - Gorlin Syndrome

QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin ...
CONTACT SUPPLIER

EsoCap AG

EsoCap AG

based inBasel, SWITZERLAND
EsoCap’s vision is to improve the lives of patients affected by esophageal diseases. The esophagus is a particularly challenging environment, as swallowed drugs only remain in the area between the mouth and the stomach for a few seconds. EsoCap, a ...
EsoCap - Drug Delivery Platform

EsoCap - Drug Delivery Platform

EsoCap owns a unique drug delivery platform allowing the topical application of drug substances for targeted and long-lasting treatment of diseases of the upper gastrointestinal tract for the first ...
CONTACT SUPPLIER

Cell Guidance Systems Ltd.

Cell Guidance Systems Ltd.

based inCambridge, UNITED KINGDOM
Cell Guidance Systems' mission is to develop products and services that enhance the human condition and protect our shared environment. We develop technologies that enable the delivery of bioactive molecules, controlling cell fate for medical, ...
CONTACT SUPPLIER

BTG International Inc.

BTG International Inc.

based inConshohocken, PENNSYLVANIA (USA)
BTG Specialty Pharmaceuticals provides rescue medicines typically used in emergency rooms and intensive care units to treat patients for whom there are limited treatment options. We are dedicated to delivering quality medicines that make a real ...
CONTACT SUPPLIER

The Medical Algorithms Company Limited

The Medical Algorithms Company Limited

based inLondon, UNITED KINGDOM
The Medical Algorithms Company supports providers and payers with analytics and documentation for evidence-based medical practice. Our platform supplies over 30,000 tested medical calculators and analytic risk scores. Nearly all specialties are ...
CONTACT SUPPLIER

Ipsen Group

Ipsen Group

based inParis, FRANCE
Ipsen is a global biopharmaceutical group dedicated to improving lives and health outcomes through innovative medicines in Oncology, Neuroscience and Rare Disease. We also have a well-established and successful Consumer Healthcare business. We are ...
CONTACT SUPPLIER

Diaceutics PLC

Diaceutics PLC

based inBelfast, UNITED KINGDOM
At Diaceutics we believe that every patient should have access to the right treatment at the right time. We provide the world’s leading pharmaceutical companies with an end-to-end solution for the launch of precision medicine diagnostics enabled by ...
DXRX - Signal Delivers

DXRX - Signal Delivers

What is DXRX Signal? DXRX Signal delivers an actionable alert of a testing behavior that allows you to engage a physician whilst they are making treatment decisions, therefore maximizing the effectiveness of your brand sales and ...
CONTACT SUPPLIER

Deep Genomics

Deep Genomics

based inToronto, ONTARIO (CANADA)
Deep Genomics recruits from among the top 1% of recent graduates and seasoned experts at the intersection of genomics, drug development and AI. Programming RNA Therapies Any Gene, Any Genetic Condition. Revolutions in AI, RNA biology and automation ...
CONTACT SUPPLIER

Pluristem Therapeutics Inc.

Pluristem Therapeutics Inc.

based inHaifa, ISRAEL
Pluristem Therapeutics Inc. is a clinical-stage regenerative medicine company using placental cells and a unique, proprietary, three-dimensional (3D) technology platform to develop cell therapies for conditions such as inflammation, muscle injuries, ...
Bone Marrow Deficiency

Bone Marrow Deficiency

Bone marrow deficiency refers to a condition in which the hematopoietic stem cells in the bone marrow fail to produce enough, or produce abnormal, red blood cells, white blood cells and platelets. Hematopoietic cell transplantation (HCT), the ...
CONTACT SUPPLIER

ICON plc

ICON plc

based inDublin, IRELAND
Our mission is to help clients accelerate the development of drugs and devices that save lives and improve quality of life. We offer a full range of consulting, clinical development and commercialisation services from a global network of offices in ...
Cell and Gene Therapies Service

Cell and Gene Therapies Service

Accelerating study start-up and delivering results with custom strategies and logistics solutions. ICON helps navigate the Cell and Gene Therapy journey by overcoming challenges and mitigating risks. Almost every cell and gene therapy clinical trial ...
CONTACT SUPPLIER

Oxford Gene Technology IP Limited

Oxford Gene Technology IP Limited

based inOxford, UNITED KINGDOM
As a leading global provider of diagnostic genomic solutions, our goal is to enable researchers and clinical decision makers to reach the right decisions for each patient, every time. The quality and innovation built into our products for NGS, ...
CONTACT SUPPLIER

HemoShear Therapeutics, Inc.

HemoShear Therapeutics, Inc.

based inCharlottesville, VIRGINIA (USA)
HemoShear Therapeutics is a clinical stage company developing novel treatments for patients with rare diseases. We are implementing a new vision for how advanced biological and computational models can accelerate the discovery of innovative ...
HemoShear - Model HST5040 - Investigational Oral Small Molecule Therapy for MMA and PA

HemoShear - Model HST5040 - Investigational Oral Small Molecule Therapy for MMA and PA

HST5040 is an investigational small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the ...
CONTACT SUPPLIER

BioMarin

BioMarin

based inSan Rafael, CALIFORNIA (USA)
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
Funding & Support

Funding & Support

We support programs and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information to the medical and scientific communities. We also support clinicians and the independent research they ...
CONTACT SUPPLIER
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