Individuals with cystic fibrosis show a notably higher rate of attention deficit hyperactivity disorder (ADHD) symptoms than the general population. This association points to a possible role of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, whose mutations cause cystic fibrosis, in the development of ADHD.

Ahead Therapeutics SL has received positive feedback from the EMA on its way toward initiating regulatory toxicology studies for its lead program in myasthenia gravis. The feedback supports the company’s scientific approach.

Stroke accounts for nearly 12% of all deaths worldwide, making it the second leading cause of death. In acute ischemic stroke, the ischemia and subsequent return of blood flow create oxidative and nitrosative stress that can overwhelm neurons and disable important circuitry.

Armatus Bio Inc.’s development of ARM-201, an AAV-delivered microRNA therapy for facioscapulohumeral muscular dystrophy (FSHD), has been boosted by a $3 million investment by Solve FSHD.

Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.

Abnormal aggregation of α-synuclein is thought to contribute to the pathology of Parkinson’s disease, the second most frequent neurodegenerative disorder. Therefore, developing drugs that prevent such aggregation could be effective for slowing or even preventing the disease.

Unravel Biosciences Inc. has submitted clinical study applications in Colombia seeking to begin proof-of-concept clinical trials for RVL-001 for Rett syndrome and Pitt-Hopkins syndrome.