Companies related to rare disease

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

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

Research...for a healthy future

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Serving Patients, Improving Lives

Science for Life

AI Accelerated Preventative Healthcare

Pharmaceutical Company & Service Partner

Advancing peptides for the treatment of rare endocrine and related diseases

Where Empathy Meets Science to Accelerate Life Sciences Research

In Pursuit of Answers

The standard of NAD measuring.

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

Your Leader in Rare Disease and Complex Therapy Programs

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

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.

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

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

Accelerating Drug, Biologic and Medical Device Clinical Development

Alone we are rare. Together we are strong.®

Better Science, Better Lives.

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

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

Enabling a better future for people with rare immune disorders

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

Quality development & manufacturing. Customized solutions.

Building a better future for everyone.

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

Going Beyond Every Day

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.

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

Better Health, Brighter Future | Santé meilleure, avenir prometteur

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

Global fieldwork management, pharma, biotech and healthcare

Rare but not alone

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

Transformation in Sight

Decoding Biology to Radically Improve Lives

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

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

FOR RARE, BY RARE

FOR RARE, BY RARE

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

Working together to find a cure

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

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

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

Bringing expertise to deliver medicines our world can trust.

Clintec is an IQVIA company.

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

Creating transformative therapies for genetic diseases that improve lives

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

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

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

Independent Thinking. Individualized Care.

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

We Connect- We Inform - We Collaborate

A transformative idea for transformative medicines

Genomic intelligence for clinical diagnostics and precision medicine development

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

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

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

Your partner for bringing science to life.

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

Rare Disease Consulting and Service Organization

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

Your Compassionate Health Ally

19 years connecting talent with life sciences companies

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

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

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

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

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

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

Rare Solutions, Extraordinary Results.

Your partner of choice for advancing rare disease research

Treating Neurodegenerative Disease by Restoring Autophagy

🔹 Premier Positions. Premier Talent. Premier Pharma. 🔹

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

Connecting Families - Raising Awareness - Building Futures

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

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

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

Changing patients' lives everyday.

International Pharmaceutical Group Certified B Corp

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

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

Patient Lifestyle and Disease Data Interactium (PaLaDIn)

Excellence in Ophthalmic Research

Pioneering Cures Through Collaborative Computing

The Evolution of Clinical Research

ESG Impact with an ROI!

Pioneering Cures Through Collaborative Computing

Unistel Medical Laboratories is a leading human and animal genetic testing centre.