Social Media Ideas for Rare And Orphan Diseases

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...

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...

‘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.

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.

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

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...

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...

Invisible Work of Rare Pharmacy Delivers Highly Visible Patient Outcomes

Invisible Work of Rare Pharmacy Delivers Highly Visible Patient Outcomes Anonymous (not verified) Fri, 08/21/2026 - 06:38 pharmagather item url Invisible W...

<![CDATA[Your Questions Answered: What This Week's Rare Disease and Specialty Drug News Tells Us About Where the Indu...

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...

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...

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...

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...

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...

An AI drug specialist zeroes in on rare forms of obesity

Superluminal Medicines banked $60 million in funding to support development of a drug designed to work similarly to — but potentially more selectively than — Rhythm Pharmaceuticals...

Invisible Work of Rare Pharmacy Delivers Highly Visible Patient Outcomes

Today's guest post comes from Jay Weaver, VP, Payer Solutions at PANTHERx® Rare. Jay describes the clinical and operational work that often happens behind the scenes at a Rare...

Ionis’ rare neurology drug approved; Pfizer inks an ADC deal

Zanvastro is now the first approved medicine for Alexander disease. Elsewhere, an immune system specialist posted positive results and a cell therapy developer turned to layoffs.

New pill provides a meaningful step forward for those fighting a rare and debilitating disease

The FDA approved a first-of-its-kind oral drug for dermatomyositis, a rare autoimmune disease. Trials showed the drug improved muscle and skin outcomes — but not without risk.

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...

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.

Rare disease drug recalled in Ireland after EMA review

A drug used to treat rare autoimmune diseases has been pulled from shelves in Ireland after it was linked to 20 deaths in Japan.

<![CDATA[BioMarin and n-Lorem Foundation Enter Research Collaboration for ReNU Syndrome Treatment ]]>

Ultragenyx Trial Failure Revives Debate About How to Treat a Rare Neuro Disorder

Ultragenyx Pharmaceuticals’ apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The di...