Social Media Ideas for Rare Disease

Rare Disease Hunger Games

Rare Disease Hunger Games Anonymous (not verified) Sun, 08/16/2026 - 03:35 field_thread_url https://cafepharma.com/boards/threads/rare-disease-hunger-games...

Rare Disease Hunger Games

Rare Disease Hunger Games Anonymous (not verified) Mon, 08/24/2026 - 06:39 field_thread_url https://cafepharma.com/boards/threads/rare-disease-hunger-games...

‘Too Rare to Care’? A New Center for Rare Diseases Hopes to Change That

A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.

Alveolar Capillary Dysplasia: How Health System Data Illuminate Rare Disease

Alveolar capillary dysplasia is one of medicine’s most devastating diagnostic puzzles: newborns appear to have lungs, but their microscopic architecture prevents those lungs from p...

Malaysia registers 26 rare disease medicines since 2020, says Health Ministry

KUALA LUMPUR, July 28 — A total of 26 medicines for rare diseases had been registered in Malaysia as of April this...

How One Woman With a Very Rare Disease Secured a Clinical Trial

Through sheer determination, Neena Nizar has become the first patient in a clinical trial testing a treatment for her disease — one so rare it affects just 30 people worldwide.

Rare TBX4 mutations reveal excess lung muscle behind childhood pulmonary hypertension

For families of children with rare diseases, the search for answers can be lonely, desperate and frustrating. Such is the case with pulmonary hypertension, or PH. Defined as high b...

RA Capital launches rare disease company Oak Hill Bio onto Nasdaq via SPAC

Rare disease biotech Oak Hill is headed to the Nasdaq through a SPAC merger with RA Capital-sponsored Research Alliance Corporation III (RACC) to fund a rare genetic disease candid...

Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline

After Jenny Pearson’s daughter Elise first had a seizure at 16 months old, an MRI came back clean—her brain “looked beautiful,” Pearson recalled to Fierce. But a follow-up scan a y...

Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare Disease

Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene ther...

Rare disease foundation partners with Citizen Health to embed AI agent into everyday care

The Angelman Syndrome Foundation is partnering with startup Citizen Health to offer families access to Ari, an artificial intelligence-powered advocate designed for rare disease co...

Podcast Series “Wheel Talk” Designed To Amplify Stories Of People Living With A Rare Disease

According to statistics shared by Rare Disease Day, a global advocacy movement lead by patients, there are 300 million people worldwide living with a rare disease. In the United St...

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.

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 ...

<![CDATA[U.S. Rare Disease Biotech Investing Rises After FDA’s PRV Program Renewal: Report ]]>

FDA Approves Ionis Pharma Drug, the First for Ultra-Rare Alexander Disease

Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experienc...

Leo Pharma buys rare skin disease drug in $435M deal

Tanabe Pharma has already asked the FDA to approve the medicine for two conditions that cause severe reactions to sunlight.

HSE approves reimbursement of treatment for patients with rare disorder

Friedreich’s ataxia is a rare, progressive, neuromuscular condition affecting about 200 people in Ireland