Social Media Ideas for Ultragenyx Pharmaceutical

<![CDATA[Ultragenyx Shares Drop Following Failed Phase III Clinical Trial ]]>

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.

Ultragenyx (RARE) Q2 2026 Earnings Call Transcript

Record revenue of $214 million signals momentum ahead of gene therapy launches.

Ultragenyx fails phase 3 Angelman test, plots ‘significant expense reductions’

Ultragenyx had been flying high after its recent FDA approval for Genglycos—but the company has been brought back down to earth with a damaging phase 3 flop.

Ultragenyx = Fraud

Ultragenyx = Fraud Anonymous (not verified) Thu, 08/13/2026 - 03:33 field_thread_url https://cafepharma.com/boards/threads/ultragenyx-fraud.647808/...

Ultragenyx to weigh ‘significant’ cost cuts as Angelman drug fails key study

The failure of what was once considered a potential blockbuster therapy represents a major setback in a tough-to-treat disease and is forcing Ultragenyx to closely examine its expe...

Ultragenyx: Angelman Study Setback Creates More Risk For Rare Disease Specialist

<![CDATA[Pharmaceutical Executive Daily: Ultragenyx's Apazunersen Fails Phase III Trial]]>

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...

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...

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

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...

Scholar Rock (SRRK) Q2 2026 Earnings Call Transcript

Apitegromab awaits FDA decision on Sept. 30 for spinal muscular atrophy treatment.

BioMarin axes $270M rare disease asset after missing phase 3 endpoint

BioMarin Pharmaceutical has ended development of BMN 401 in all indications months after reporting mixed phase 3 data on the rare disease prospect.

UniQure, after setbacks, seeks FDA approval of Huntington’s gene therapy

The company is now hoping for an affirmative answer on its application after an unusually turbulent year dealing with U.S. regulators.

Unicycive Therapeutics: Two CRLs, One Fixable Problem

Novartis RNA drug falls short in rare muscle disease study

The failure of “del-desiran,” which Novartis got through a $12 billion acquisition, could raise questions about the company’s business development acumen, according to one analyst.

BioMarin to acquire Alesta Therapeutics for up to $490 million

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

Epicrispr lands $90M to advance epigenetic editing drug for rare muscle disease

The startup has completed enrollment of an early-stage study testing a first-of-its-kind treatment for facioscapulohumeral muscular dystrophy, a disease targeted by many other drug...

Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings

The FDA has put a clinical hold on Regenxbio’s RGX-121 over asymptomatic spine MRI findings, derailing the biotech’s plans to refile for approval of the once-rejected gene therapy...

Crystalys nets $130M more to push gout drug through late-stage tests

The startup has a broad global development program underway — and the support of more than 20 investment firms — for a treatment that’s approved in many Asian countries but isn’t y...

Argenx jumps on Phase 3 data for blockbuster Vyvgart

The latest results give investors confidence in the drug's ability to be approved in another indication for which there are no approved treatments. 

Zydus’ Sentynl pens $475M deal for Mereo’s phase 3-ready rare genetic lung disease drug

Sentynl Therapeutics has penned a deal potentially worth $475 million for the option to market Mereo BioPharma's phase 3-ready rare genetic respiratory disease drug in the U.S.