Company will host a webcast at 10am ET on November 6, 2025, to discuss its Phase 2a study to assess efficacy, safety and ...

An update from Bright Minds Biosciences ( ($TSE:DRUG) ) is now available. Bright Minds Biosciences has announced the initiation of its ...

Rhythm Pharmaceuticals (($RYTM)) announced an update on their ongoing clinical study. Study Overview: Rhythm Pharmaceuticals is conducting a Phase ...

US biotechs Acadia Pharma and Harmony Biosciences have both suffered setbacks in phase 3 trials of drugs for rare diseases – respectively Prader-Willi and Fragile X syndrome, and in one case at least, ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy and safety of GTX-102 (apazunersen), an ...

Ultragenyx Pharmaceutical has dosed the first participant in Aurora, a study evaluating GTX-102 (apazunersen) for angelman ...

Mitch McKenny ran from Broken Hill to Newcastle to raise awareness for Angelman syndrome. Medical staff in Dubbo implored him to stop running when he began experiencing pain in his hip, but he would ...

ACTORS Colin Farrell and Jai Courtney have joined forces with other Australian identities to spread awareness about a rare genetic condition in a video and social media campaign. Farrell’s son has ...

This has sparked a debate on social media about a condition something known as the ‘Six Pocket Syndrome’. A teacher shared a video, pinning the bad attitude of single kids to the many people in their ...

A KBC contestant's confident demeanor sparked debate, with many labeling him "spoiled." Psychologists explain this as "six pocket syndrome," where overindulgence, not being an only child, shapes ...

Angelman syndrome is a rare genetic, nondegenerative and neurodevelopmental disorder caused by mutations affecting the expression of maternal UBE3A, which is expressed in neurons and is a key protein ...

For decades, the diagnosis of shaken baby syndrome has been putting parents in jail all over the world. But some jurisdictions are beginning to ask: are heartbroken people being put in the dock for ...