Chicago, July 2025 – New research from the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago reveals groundbreaking insights into how mitochondria, ancient bacterial descendants within our cells—may be key contributors to two distinct childhood neurological disorders: spinal muscular atrophy (SMA) and autism spectrum disorder (ASD).
By CNKE News Desk | Published in Nature Communications
In a promising development for children affected by Rett syndrome, researchers led by Dr. Sanchita Bhatnagar at UC Davis Health have created a novel gene therapy that reactivates silenced healthy genes. This therapeutic approach could potentially treat Rett syndrome and other X-linked conditions like fragile X syndrome.
Read more: Breakthrough Gene Therapy Offers Hope for Rett Syndrome
Washington, July 18, 2025 – Sarepta Therapeutics has come under severe scrutiny following reports that the U.S. Food and Drug Administration (FDA) is requesting a voluntary suspension of all shipments of Elevidys—its AAVrh74-based gene therapy for Duchenne muscular dystrophy (DMD). This move follows three deaths possibly associated with Sarepta’s gene therapy platform, including two teenage DMD patients and one adult with limb-girdle muscular dystrophy (LGMD).
Breakthrough in Mitochondrial Donation IVF: Eight Healthy Babies Born Following UK Pronuclear Transfer Treatment
Newcastle, UK – A pioneering IVF technique designed to prevent the transmission of mitochondrial DNA disease has resulted in the birth of eight healthy babies, according to landmark studies published in The New England Journal of Medicine.
GLP-1 Drugs Show Promise in Treating Idiopathic Intracranial Hypertension: A New Frontier in IIH Management
In a major development for the treatment of Idiopathic Intracranial Hypertension (IIH), a large retrospective cohort study from the TriNetX US Collaborative Network suggests that GLP-1 receptor agonist (GLP-1 RA) therapy may significantly improve outcomes compared to traditional treatments such as acetazolamide and topiramate.
Read more: GLP-1 Drugs Show Promise in Treating Idiopathic Intracranial Hypertension
CHICAGO — A new Northwestern Medicine study provides compelling evidence that may shift the narrative surrounding youth contact sports and brain health. Contrary to longstanding concerns, researchers found no increase in phosphorylated tau (p-tau) protein in a key brain region among former high school and college football players.
Read more: New Study Questions Link Between Youth Football and Brain Tau Protein
A recent experimental study published in LabMed Discovery has found that a nanoemulsion formulation of vitamin D3 significantly improves core symptoms of autism spectrum disorder (ASD) in young children. The research offers a new direction in nutritional interventions for neurodevelopmental disorders.
Read more: Vitamin D3 Nanoemulsion Shows Promise in Improving Core Symptoms of Autism
In a groundbreaking study published in Nature on July 9, 2025, researchers at NYU Langone Health reported remarkable progress in treating HPDL deficiency—a rare mitochondrial disease—using an experimental therapy. The condition impairs the production of coenzyme Q10 (CoQ10), vital for cellular energy.
Read more: Experimental Treatment Offers Hope for Children with Rare Mitochondrial Disorder
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