Social Media Ideas for Rare Disease

Is Rare Disease a good place to be?

Is Rare Disease a good place to be? Anonymous (not verified) Tue, 06/23/2026 - 00:01 field_thread_url https://cafepharma.com/boards/threads/is-rare-disease...

Is Rare Disease a good place to be?

Is Rare Disease a good place to be? Anonymous (not verified) Thu, 07/02/2026 - 06:06 field_thread_url https://cafepharma.com/boards/threads/is-rare-disease...

Father of rare disease patient fears losing 'second son'

Patients with rare diseases and their families travelled to Leinster House to call for action on access to new medicines.

When diagnosing rare disease falls to the family

Medicine tells a clean story about diagnosis. A set of symptoms appears. A clinician evaluates them. Tests are ordered. Patterns emerge. A name is assigned. Uncertainty resolves. T...

<![CDATA[The New Capital of Care in Rare Disease]]>

Regeneron Cancer Drug Shows New Promise in a Rare Disease… and Maybe More

Regeneron Pharmaceuticals’ multiple myeloma drug linvoseltamab has clinical trial results showing complete responses in 90% of patients who have light chain amyloidosis, a rare blo...

A genome-first multiomic approach to diagnosis

Did you know that one in 10 Americans have a rare disease and it can take five or more years, on average, for a person with a rare disease to get an accurate diagnosis? That’s more...

Living with cystinosis: The rare disease that upended my life, but taught me resilience

Lucy Beckwith was diagnosed with a rare condition as a baby and has since faced kidney failure, spinal surgery and a transplant in her teens.

The Orphanet Nomenclature and Classification of Rare Diseases for Improved Patient Recognition and Data Interoperability...

Background: Although individually uncommon, rare diseases (RDs) collectively affect an estimated 329-624 million people worldwide. There are over 6500 known RDs, 85% of which affec...

3 Kentucky sisters diagnosed with ultra-rare disease as parents fight to raise awareness

TBCK is an extraordinarily rare genetic condition. Dr. Dustin Wells of Wayne County Hospital said there are only approximately 170 confirmed cases in the world.

Garetosmab may benefit patients with rare bone disease

CHICAGO — Garetosmab reduced new heterotopic bone lesions in patients with fibrodysplasia ossificans progressiva at 56 weeks compared with placebo, according to results of the OPTI...

<![CDATA[The Regulatory Landscape for Rare Pediatric Diseases]]>

This New DNA Test Solves Rare Disease Mysteries That Standard Genetics Misses

A new long-read DNA test improves rare disease diagnosis, replaces multiple existing tests, and could become the preferred global standard for genetic diagnostics. A new DNA test i...

Gene therapy shows promise in ARC syndrome, a deadly childhood liver disease

A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hosp...

Sanofi and Wave post AATD updates in race to end genetic disease’s 40-year new treatment drought

Sanofi and Wave Life Sciences are using their time at the annual meeting of the American Thoracic Society in Orlando to share updated data for their next-gen attempts to treat a pr...

<![CDATA[Addressing Prohibitive Costs for Rare Diseases]]>

<![CDATA[Finding the Data for Rare, Pediatric Therapies: Q&A with Lisa Bollinger]]>

Genomic tool highly effective at detecting rare disease diagnoses

A newly developed open-source tool designed for rigorous reanalysis of genomic data is highly effective at detecting new rare disease diagnoses. The tool's ability to frequently an...

In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy

In another sign that the FDA is easing its stance toward rare disease drug candidates under new leadership, the agency will reconsider Regenxbio’s gene therapy for an ultra-rare ch...

Relay drug shows early promise against rare blood vessel diseases

While early, the findings suggest the therapy could be superior to Novartis’ Vijoice at treating “vascular anomalies,” a cluster of chronic conditions with few available treatments...

Pike Co. Toddler Diagnosed with Rare Condition

A two-year-old Pike County girl is the first known child in the U.S. diagnosed with an extremely rare genetic disorder called Rajab Syndrome. Doctors ...

Hurdles persist for persons with rare diseases seeking disability certification

Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD

Elixirgen's lead program is an ex vivo autologous therapy based on proprietary tech that targets telomere biology disorders, rare genetic diseases characterized by short telomeres...

1 in 5 Parkinson’s cases may be a different disease

Each year in the US, around 90,000 people are diagnosed with Parkinson’s disease.Continue ReadingCategory: