Companies related to Rare disease

We Empower the Life Science, FMCG, and Financial industries with innovative solutions.

Advancing peptides for the treatment of rare endocrine and related diseases

Serving Patients, Improving Lives

Baylor Genetics pioneered the history of genetic testing. Now, we’re leading the way in precision medicine.

This page is no longer active. Please follow us at Labcorp Drug Development.

Our mission is to treat and cure FA by marshaling and focusing global resources and relationships.

Science for Life

AI Accelerated Preventative Healthcare

Pharmaceutical Company & Service Partner

Providing Pharma & Biopharma with RWD & AI-Driven Insights on Patient Populations Affected by Speciality & Rare Diseases

Empowering Health: Top Tier Pharmaceutical Distribution since 1952 - Serving the Jordanian Market with Excellence

In Pursuit of Answers

Alone we are rare. Together we are strong.®

Your Leader in Rare Disease and Complex Therapy Programs

An integrative analytic platform connecting phenotypes to genotypes across species, bridging basic and applied research.

The standard of NAD measuring.

Research...for a healthy future

As a leading full-service global CRO, we work to create solutions that advance new treatments from discovery to reality.

As a leading full-service global CRO, we work to create solutions that advance new treatments from discovery to reality.

Where Empathy Meets Science to Accelerate Life Sciences Research

Accelerating Drug, Biologic and Medical Device Clinical Development

Better Science, Better Lives.

MCRI is dedicated to making discoveries to prevent and treat common and rare childhood conditions.

The Premier Rare Disease Company in Israel. We Deliver Rare Dedication to Wellness.

Unsere Philosophie ist das Schlüssel-Schloss-Prinzip: für langfristig erfolgreiche Kunden- und Kandidatenbeziehungen.

Full-Service CRO delivering excellence in early stage clinical research, from Phase I & HAL trials to BE/BA studies.

Quality development & manufacturing. Customized solutions.

Providing relief to patients with unmet medical needs in select specialty and rare diseases.

Enabling a better future for people with rare immune disorders

Global fieldwork management, pharma, biotech and healthcare

Going Beyond Every Day

A biopharmaceutical company focused on delivering Medicines that Make a Difference® in patients’ and caregivers’ lives.

Better Health, Brighter Future | Santé meilleure, avenir prometteur

Building a better future for everyone.

Transformation in Sight

Rare but not alone

We’re Built for Biotech℠, dedicated to helping today’s top innovators turn big ideas into life-changing treatments.

Through research, technology and collaboration, we make medical breakthroughs for those with NMOSD. Next up: CURES.

Leading patient advocacy organization working to eliminate the challenges of rare disease. #CareAboutRARE #RareDisease

Clintec is an IQVIA company.

Leading the fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases.

FOR RARE, BY RARE

FOR RARE, BY RARE

Support + Educate + Advocate - Preemies & Pros from Pregnancy to the Adult Years

PNRI unravels the powerful mysteries of genetics to drive future medical breakthroughs.

Leading patient advocacy organization working to eliminate the challenges of rare disease. #CareAboutRARE #RareDisease

Decoding Biology to Radically Improve Lives

Transforming healthcare connections

Great Science Deserves Great People.

Great Science Deserves Great People.

Great Science Deserves Great People.

AI for Faster Rare Disease Diagnosis | Scalable PaaS Solutions for Hospitals & Pharma

Clinical research value chain organization (trial management, SMO, warehousing, supply, sourcing and distribution)

Finding Innovative Global Health Solutions For Today and Tomorrow Through Clinical Research. # OrganicCRO

Bringing expertise to deliver medicines our world can trust.

Kyowa Kirin International | Making people smile | www.KyowaKirinInternational.com

Creating transformative therapies for genetic diseases that improve lives

Developing best-in-class curative gene therapies for rare disorders.

A transformative idea for transformative medicines

Clinical-stage lentiviral gene therapy company, powered by plato®. Our vision: bring personalized gene therapy worldwide

Leading European Umbrella Association for Pulmonary Hypertension | Join Us in Empowering PH Patients!

Working together to find a cure

Genomic intelligence for clinical diagnostics and precision medicine development

A relentless force in finding a cure and improving the lives of people affected by scleroderma.

Bringing life changing treatments to people with genetic disease, regardless of prevalence or commercial interest

Rare Disease Consulting and Service Organization

Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases.

Independent Thinking. Individualized Care.

Komtur delivers comprehensive healthcare solutions that connect pharmaceutical innovations with patients.

Your partner for bringing science to life.

Global Multi-Award-Winning Access Experts for Orphan Drugs, Cell and Gene Therapies

19 years connecting talent with life sciences companies

We push the boundaries of science to deliver life-changing medicines.

Your Compassionate Health Ally

Providing solutions to better navigate the inherent challenges of rare and specialty disease

Serving patients across Middle East, North Africa & Turkey through collaborative partnerships.

Rare Solutions, Extraordinary Results.

Clinical Artificial Intelligence to improve diagnosis, management and research of Rare and Genetic Disease

Die Schweizerische Neurologische Gesellschaft ist der Berufsverband der Fachärztinnen und Fachärzte für Neurologie

Treating Neurodegenerative Disease by Restoring Autophagy

We Connect- We Inform - We Collaborate

Global "networked" consultancy specializing in customer engagement & digital transformation in Health + Life Sciences.

Your partner of choice for advancing rare disease research

Helping healthcare companies envision the future by generating customer insights to guide commercial success

STAR, a 501(c)3 non-profit, is dedicated to Salla disease research, treatment, education, awareness & family networks.

🔹 Premier Positions. Premier Talent. Premier Pharma. 🔹

Connecting Families - Raising Awareness - Building Futures

Changing patients' lives everyday.

On a mission to improve your experience living with a chronic health condition

International Pharmaceutical Group Certified B Corp

An innovative gene editing company discovering and developing curative treatments for genetic diseases

The world’s largest research-focused patient advocacy organization for facioscapulohumeral muscular dystrophy (FSHD) .

Pioneering Cures Through Collaborative Computing

Pioneering Cures Through Collaborative Computing

Kyowa Kirin North America is a specialty pharmaceutical company dedicated to making a profound impact on patient lives.

Patient Lifestyle and Disease Data Interactium (PaLaDIn)

Excellence in Ophthalmic Research

Your One Answer Genetic testing for rare diseases made more efficient and more accessible

We are a late-stage biopharmaceutical company focused on developing & commercializing products to treat rare diseases

ESG Impact with an ROI!

Franklin harnesses the power of its community to enable actionable insights, serving professionals at the point of care