Diseases
Research published in Pediatric Blood & Cancer has found that the medication Abatacept prevents severe acute graft-versus-host disease (GVHD) without increasing the incidence of graft failure in pediatric patients with bone marrow failure syndromes. A study conducted by researchers at Cincinnati Children's Hospital Medical Center involved 26 patients
Diseases
Lundbeck's pipeline developments for amlenetug, a novel investigational molecule for treating Multiple System Atrophy (MSA), will be presented at the 2025 International Congress of Parkinson's Disease and Movement Disorders in Honolulu, Hawaii. Amlenetug, a human monoclonal antibody, targets pathological aggregations of the protein a-synuclein in the
Diseases
Shape Therapeutics, a leader in RNA-based gene therapy, has presented new preclinical data showcasing the therapeutic potential of its lead product, SHP-201, a disease-modifying gene therapy for the treatment of Parkinson's Disease (PD). The Company also presented data highlighting its novel BBB-penetrant, engineered AAV5 capsid, SHP-DB1, which efficiently
Diseases
Abeona Therapeutics Inc., a commercial-stage biopharmaceutical company, has announced that its ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected for the U.S. Food and Drug Administration's (FDA) Rare Disease Endpoint Advancement (RDEA) Pilot Program. This program aims to support the development and timely approval of
Diseases
The Grand Challenges in Parkinson's Disease symposium and Rallying to the Challenge meeting brought together hundreds of scientists, clinicians, and people with Parkinson's to explore the latest research and accelerate research in the field. The meeting highlighted the role of the endolysosomal pathway in disease, and
Child development
A recent study published in the Journal of Family Psychology has confirmed the theoretical utility of the family stress model (FSM) across three common family structures in the United States: biologically connected/adopted two-parent families (T-B/A), stepfamilies (ST), and single-parent families (SP). The study, conducted by researchers at the
Diseases
Research has shown that mental health disorders are concerningly high among young people, with one in five beginning in early childhood. A study examining the developmental trajectories of internalising and externalising symptoms in young Australians identified social determinants that predict these psychiatric symptom trajectories. The study found that reducing maternal
Diseases
A long-held belief that weight loss is the primary way to lower disease risk is being challenged by new research, which reveals that prediabetes can go into remission without weight loss, offering a significant shift in how doctors treat patients at high risk for diabetes. The study, published in Nature
Diseases
Mayo Clinic researchers have identified a key enzyme called valosin-containing protein (VCP) that plays a crucial role in recycling damaged proteins in the liver. When exposed to excessive alcohol, VCP becomes nearly completely removed from the lipid droplet surface, allowing a specific protein called HSD17b13 to accumulate, leading to fatty
Diseases
Researchers at Sutter Roseville Medical Center in Roseville, California, have reported a case of a 44-year-old African American female who developed anti-glomerular basement membrane (anti-GBM) disease after contracting COVID-19. The patient presented with acute kidney injury, hematuria, and shortness of breath, and despite aggressive therapy, renal recovery was not achieved.
Diseases
Austria's Alzheimer Society conducted a survey of hospital-based departments of neurology and psychiatry to assess their readiness for disease-modifying therapies with amyloid-antibodies for patients with early Alzheimer's disease. The research, led by Gerhard Ransmayr, Medical Faculty, Johannes-Kepler-University, found that many centers lack the necessary resources, including
Diseases
MapLight Therapeutics has initiated a Phase 2 study to evaluate the efficacy, safety, and tolerability of ML-007C-MA, an investigational novel M1/M4 agonist, as a potential treatment for Alzheimer's disease psychosis (ADP). The study, known as the VISTA study, has been designed based on the results of an
