LUCY Glenn appeared “completely healthy” until she was 18 months old when her behaviour suddenly changed. Her mum, Katherine McCready, 31, said noticed she suddenly stopped playing ...

In the past year, an unusual frequency of gallbladder disease was recognized in girls or women with Rett syndrome (Kathy Hunter, International Rett Syndrome Association, personal communication ...

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

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

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

The International Rett Syndrome Foundation (IRSF), headquartered in Cincinnati, has announced the allocation of nearly $2 million in grants to seven leading researchers, ...

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

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

Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med -- Study supported FDA and Health Canada ...

Decision on orphan drug designation for NTI164 to treat Rett syndrome expected in ‘due course’ US Food and Drug Administration granted orphan designation for NTI164 in Rett syndrome in November 2024 ...