Social Media Ideas for Orphan Drugs

‘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.

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...

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...

<![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...

Regeneron to challenge Ipsen as FDA clears bone disease drug

Pasatru’s approval sets up a market battle in a rare indication where the only available treatment has generated disappointing sales.

Non-Profit Pharmaceutical Manufacturer Delivers Drug Access to Rural Hospitals

BioMarin acquires experimental drug for rare bone disorder in $275M deal

The Alesta buyout helps restock BioMarin's pipeline with an asset it says could “reach our largest addressable patient population,” joining other muscle and skeletal disease treatm...

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.

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...

<![CDATA[FDA Approves Zanvastro for Alexander Disease in Pediatrics & Adults ]]>

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...

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...

<![CDATA[Pharmaceutical Executive Daily: BioMarin and n-Lorem Foundation Enter Collaboration]]>

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.

FDA OKs Takeda, Protagonist drug for rare blood cancer

The clearance and broader-than-expected label for Mimrylo open up a yearly revenue opportunity for Takeda that some analysts expect to reach $2 billion or more. 

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...

First-in-Class Takeda, Protagonist Drug Lands FDA Approval in Rare Blood Disorder

Takeda Pharmaceutical’s Mimrylo is now approved for treating polycythemia vera, a rare blood cancer with limited therapeutic options. Originally developed by Protagonist Therapeuti...

PTC Therapeutics’ $211M Bid Wins Bankruptcy Auction for Sangamo Gene Therapy

Sangamo Therapeutics filed for Chapter 11 bankruptcy in June after its search for strategic alternatives failed to find a path forward for the company. PTC Therapeutics’ auction wi...

Ultragenyx, after setbacks, nabs first gene therapy approval

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

‘We’re moving quickly’: Startup bags rare disease drug as Thiel-backed CEO eyes $50M fundraise

A rare disease startup with a mission to repurpose proven drugs has licensed a Chinese formulation of a well-known medicine and set its sights on a $50 million fundraise.

<![CDATA[FDA Approves Pasatru For Adults With Fibrodysplasia Ossificans Progressiva ]]>

Amylyx’s bet on a GLP-1 blocker pays off

A late-stage trial found the drug, which Amylyx bought for cheap in a bankruptcy auction, was effective at regulating blood sugar in patients who’d just undergone bariatric surgery...