Robuta

Apr 10, 2025 - Of the 7,000 known rare diseases, less than 10% have an available treatment option. Since 2007, through our dedicated business unit Recordati Rare Diseases,...

We’re advancing innovative treatments in rare diseases and blood disorders, leveraging RNAi, nanobody technology, and enzyme replacement therapies to address...

Jan 24, 2025 - Rare Diseases International is the global alliance of Persons Living with a Rare Disease and their families.

Our scientists use the latest technologies to develop and deliver therapies that are used to treat rare and serious conditions, including immunodeficiency and...

CSL Behring is committed to improving the quality of life for people with rare and serious diseases worldwide.

A global media and technology company.

Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Learn more about us here.

Dec 17, 2025 - Duchenne muscular dystrophy and metachromatic leukodystrophy were added to the list of conditions the government recommends states screen newborns for. 

Our scientists use the latest technologies to develop and deliver therapies that are used to treat rare and serious conditions, including immunodeficiency and...

May 27, 2025 - Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this technique help to unlock...

Dec 2, 2025 - IndoUSrare is a non-profit organization, supports rare disease research, awareness, and advocacy, fostering impactful global collaborations for solutions.

The death of Prince Frederik of Nassau, at the age of 22, has caused deep sadness in the Grand Duchy of Luxembourg, but also among citizens worldwide who have...

Researchers at UCL and Great Ormond Street Hospital (GOSH) have developed the first-ever lab-grown mini-stomach that contains the key components of the...

Nov 19, 2025 - At two upcoming events, experts will offer practical solutions to key challenges for oncology and rare disease clinical trials.

popEVE, an AI tool developed by Harvard researchers, classifies genetic variants by disease potential, aiding in the diagnosis of rare genetic disorders.

Some autoimmune diseases can be fatal. Learn about four serious types and their symptoms, treatments, and long-term outlooks.

May 8, 2024 - EveryLife Foundation Releases New Study Revealing Economic Burden of Rare Diseases in the U.S. Approached $1 Trillion in 2019 By Britta Dornan, Senior...

CSL Behring is proud to serve the rare and serious diseases community with treatments in numerous therapeutic areas.

Sep 11, 2025 - Genethon develops innovative therapies for rare diseases of the eye, the liver, the immune system and for neuromuscular diseases.

Nov 3, 2025 - The PPD™ clinical research business of Thermo Fisher Scientific advances oncology and hematology rare disease trials through extensive experience,...

The Blue Cross Blue Shield Association technology review organization, known as Evidence Street, issued a positive review of Whole Genome Sequencing for...

A Global Advocacy Network for Families with Rare Diseases and the Professionals Who Serve Them

Collectively, rare diseases affect millions, yet patients often face a

Nov 13, 2025 - Taking place in February, which also marks the occasion of Rare Disease Day, our annual EURORDIS Black Pearl Awards celebrate the outstanding achievements of...

Nov 26, 2025 - The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place,...

Jan 30, 2025 - Meet Luke and Kim, parents and two rare disease patient leaders who are accelerating groundbreaking research to find a treatment and cure for their families.

Sep 10, 2025 - A disease is considered rare when it affects fewer than 1 in 2,000 people. However, more than 6,000 rare diseases have been identified, and they collectively...

Stealth BioTherapeutics’ FDA win could help build momentum in the broader rare disease space.

Explore the challenges faced by Indian pharma in developing rare disease drugs and potential solutions for innovation and cost reduction.

Jul 9, 2025 - For a healthy Belgium: indicators of health and care

Sep 9, 2025 - Meet Jeremiah - For rare diseases, time is of the essence | Greenwood Genetic Center

Women who have the bleeding disorder often go undiagnosed, Duke University doctor says.

Jul 31, 2025 - An estimated two million Australians live with a rare disease. Approximately 80% of rare diseases are genetic disorders.

Feb 4, 2026 - For people living with rare genetic bone disorders, everyday life can be unpredictable. A minor injury, a routine dental visit, even normal growth can trigger...

Nov 19, 2025 - At two upcoming events, experts will offer practical solutions to key challenges for oncology and rare disease clinical trials.

Explore Regeneron’s research and advancements in rare diseases for conditions like FOP and cryopyrin-associated periodic syndromes (CAPS).

Join this Community The Rare Disease Community extends and generalises the system of access authorisation and high volume secure data transfer developed within...

Accelerate your fight against rare diseases with a tailored approach backed by IQVIA's industry-leading data, analytics, technology and deep expertise.

Nov 5, 2025 - We are driving a mindset shift regarding rare diseases that pose unique challenges to patients, their families, society, healthcare professionals, and...

Jan 22, 2026 - How AI and cloud technology could help speed up medical diagnoses

We’ve prioritized inclusivity and equity in health care by creating space where individuals and families impacted by rare diseases feel seen, heard and...