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) Sat, 10/03/2026 - 04:06 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) Wed, 10/07/2026 - 04:08 field_thread_url https://cafepharma.com/boards/threads/is-rare-disease...

Probably Genetic raises $12M to scale rare disease dataset, support drug discovery

The company’s core product is a physician-ordered genetic test for undiagnosed rare disease patients, who typically face a seven-year diagnostic odyssey before getting answers.

<![CDATA[Finding the Few: Combining Clinical Insight, Data and Media to Reach Patients with Rare Diseases]]>

Seltene Erkrankung: Eine Einzige von 83 Millionen

Die sechsjährige Caroline leidet am PUS3-Syndrom - als einzig bekannte Betroffene in Deutschland. Ihre Eltern haben wenig Hoffnung auf Besserung – aber einen Plan. mehr...

<![CDATA[FDA Launches Rare Disease Framework Initiative ]]>

FDA, Duke-Margolis workshop to examine evidence standards for rare diseases

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...

Cherokee Nation leaders expand rare disease care and advocacy

The Cherokee Nation held its third annual Rare Disease Summit to advance policy, care, and early intervention initiatives for tribal citizens affected by rare conditions.

Phase 3 Data Could Make Roche Drug a Contender to Treat Rare Kidney Disease`

Roche reported the drug sefaxersen achieved statistically significant and clinically meaningful results in a Phase 3 test in immunoglobulin A nephropathy, or IgAN. This antisense o...

Major breakthrough in treatment for ultra-rare 'second skeleton' disease

Patients grow extra bones where they don’t belong, including within muscles and tendons

Rare Disease Refresh: FDA’s Rare Disease Framework Initiative

By James E. Valentine & Charles G. Raver & Frank J. Sasinowski —

<![CDATA[Solving the Rare Disease Launch Paradox: Using Pre-Launch Planning and Partnership to Create]]>

Ultragenyx Gene Therapy Is First Approved Treatment for Rare Neuro Disease Sanfilippo

Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s...

'One in a million' — the devastating brain disease my dear friend died from in months

Sporadic CJD is the most common form, but variant and familial types also affect humans.

Shionogi pays $2B to acquire a rare disease startup

The acquisition of Texas-based IntraBio adds to a growing rare disease business for Shionogi by handing the company a marketed treatment for two inherited conditions.

Disc’s once-spurned rare disease drug to get a second chance at approval

Months after the FDA rejected its treatment for a rare light-sensitivity disease, the biotech claimed success in a Phase 3 trial and now plans to re-engage regulators.

Rare disease affects 5 in China after eating fish

A rare disease sickened five people in China earlier this year after they ate wild caught freshwater fish.In April 2026, five suspected Haff disease cases were reported to Longchua...

Rare Parkinson’s form more common in Pacific communities

A form of Parkinson's disease once considered rare is far more common in Pacific communities than previously recognised.

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...

Inherited lupus can begin early—and continue silently

A multicenter study led by researchers at Sultan Qaboos University has found that patients with a rare inherited form of lupus may continue to accumulate organ damage even when con...

Shionogi Grows Rare Disease Portfolio with $2B IntraBio Acquisition

Shionogi signaled its intent to focus on rare disease drugs in April when it acquired Radicava (edaravone), an amyotrophic lateral sclerosis (ALS) treatment, from Tanabe Pharma for...