Companies related to Rare Disease

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

Where Empathy Meets Science to Accelerate Life Sciences Research

Serving Patients, Improving Lives

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

AI Accelerated Preventative Healthcare

Pharmaceutical Company & Service Partner

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

In Pursuit of Answers

The standard of NAD measuring.

Alone we are rare. Together we are strong.®

Advancing peptides for the treatment of rare endocrine and related diseases

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.

Your Leader in Rare Disease and Complex Therapy Programs

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.

Science for Life

Accelerating Drug, Biologic and Medical Device Clinical Development

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

Quality development & manufacturing. Customized solutions.

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

Better Science, Better Lives.

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

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

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

Building a better future for everyone.

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

Global fieldwork management, pharma, biotech and healthcare

Better Health, Brighter Future | Santé meilleure, avenir prometteur

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

Enabling a better future for people with rare immune disorders

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

Going Beyond Every Day

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

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

Decoding Biology to Radically Improve Lives

FOR RARE, BY RARE

FOR RARE, BY RARE

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

Transformation in Sight

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

Rare but not alone

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

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

Working together to find a cure

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.

A transformative idea for transformative medicines

Creating transformative therapies for genetic diseases that improve lives

Transforming healthcare connections

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

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

Independent Thinking. Individualized Care.

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

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

Genomic intelligence for clinical diagnostics and precision medicine development

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

Rare Disease Consulting and Service Organization

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

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) .

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

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

We Connect- We Inform - We Collaborate

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

Your Compassionate Health Ally

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

19 years connecting talent with life sciences companies

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

Connecting Families - Raising Awareness - Building Futures

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

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

Treating Neurodegenerative Disease by Restoring Autophagy

🔹 Premier Positions. Premier Talent. Premier Pharma. 🔹

Your partner of choice for advancing rare disease research

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

Changing patients' lives everyday.

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

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

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

International Pharmaceutical Group Certified B Corp

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

The Evolution of Clinical Research

Patient Lifestyle and Disease Data Interactium (PaLaDIn)

Pioneering Cures Through Collaborative Computing

Pioneering Cures Through Collaborative Computing

Excellence in Ophthalmic Research

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

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