Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Suppliers & Manufacturers

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 ...

Curome Biosciences is an innovative R&D company developing therapies to treat mitochondrial dysfunction-related diseases with high unmet medical needs. Curome Biosciences wants to provide solutions for treatment of diseases with high unmet medical ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

We are a global healthcare company, founded in 1923 and headquartered just outside Copenhagen, Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity, and rare blood and rare endocrine diseases. ...

We are a clinical-stage biotechnology company pioneering the treatment of cancers with a technology platform that allows the direct delivery of immunotherapeutic payloads within the tumor microenvironment. Intrinsic resistance significantly reduces ...

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 ...

At Horizon, we believe science and compassion must work together to transform lives. Our mission to deliver medicines for rare, autoimmune and severe inflammatory diseases and provide compassionate support comes from our strong and simple philosophy ...

AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from ...

We are a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of best-in-class novel nanomedicines that combine our proprietary lipid-assembled drug delivery platform with approved active pharmaceutical ...

Oligomerix is a biotechnology company focused on developing disease-modifying and treatment therapeutics for neurodegenerative diseases characterized by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy ...

Rady Children’s Institute for Genomic Medicine is a non-profit research organization embedded within Rady Children’s Hospital-San Diego. We pioneered a medical revolution to end the diagnostic odyssey for neonatal and pediatric rare disease. Now ...

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 ...

Camurus is committed to developing and commercializing innovative and long-acting medicines for the treatment of severe and chronic conditions, including opioid dependence, pain, cancer and endocrine disorders. New drug products are based on our ...

Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases. The Protara team prioritizes creativity, diverse perspectives and tenacity to expedite our goal of bringing life-changing therapies ...

Confo Therapeutics’ unparalleled technology stabilizes functional conformations of GPCRs (G protein-coupled receptors) to uncover a wide range of previously inaccessible GPCRs as drug targets. This technology platform combined with the pharmacologic ...