Axiomer ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expect ...

Amy’s daughter, Ashley, who was diagnosed with Rett syndrome in 2015, is the inspiration behind her drive to help find a cure ...

Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF) and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively ...

Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of ...

Learn more about whether ACADIA Pharmaceuticals Inc. or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ ...

A delivery driver has quit his job to run the length of the UK in a month for a cause close to his heart. Jack Ward, 24, from ...

What is Guillain-Barré syndrome? Guillain-Barré syndrome (GBS) is also called acute inflammatory demyelinating polyradiculoneuropathy (AIDP). It's a neurological disorder in which the body's immune ...

NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.

Heading the BNA2025? We've gone through the scientific program to find the sessions we think the BioTechniques audience will ...

Healthcare analysts believe that shares in rare neurological share play Neuren Pharmaceuticals are well undervalued after a ...

Reports FY24 revenue $8.333M vs $15.451M last year. “We are pleased with the pace at which our TSHA-102 clinical program is advancing across a ...

Endres dedicated the game-winning goal to his sister, Jillian, who passed away in December 2024 due to complications from Rett syndrome. The Dolphins will play second-seed Hanover on Wednesday at ...