Tag

Therapies

All articles tagged with #therapies

Normal for 18 months, then Rett syndrome: a family's long road to a diagnosis
health1 month ago

Normal for 18 months, then Rett syndrome: a family's long road to a diagnosis

A father recounts how his daughter appeared perfectly normal for the first 18 months, then rapidly regressed in speech and motor skills, leading to a long diagnostic journey that culminated in Rett syndrome in October 2022; after initial ER visits and a misdiagnosis of autism, genetic testing confirmed Rett, and now five-year-old Talia receives multiple therapies with the family hopeful for FDA-approved treatment and continued support.

Decoding Alzheimer's Mutation: Unique Brain Plaques Unveiled
health1 year ago

Decoding Alzheimer's Mutation: Unique Brain Plaques Unveiled

Researchers have decoded the structure of amyloid beta fibrils linked to the Arctic mutation, a rare inherited form of Alzheimer's disease, using cryo-electron microscopy and NMR. This mutation leads to the formation of unique cottonwool plaques, which are significantly larger than typical Alzheimer's plaques. Understanding these structures could guide the development of targeted therapies, particularly antibody-based treatments, for specific Alzheimer's subtypes.

Emerging Therapies Spark Interest in Rare Cardiovascular Disease
healthcare2 years ago

Emerging Therapies Spark Interest in Rare Cardiovascular Disease

The global market for pulmonary arterial hypertension (PAH) treatments is expected to reach nearly $11 billion by 2030, attracting the interest of biopharma companies. PAH is a rare cardiovascular disease characterized by the thickening and narrowing of arteries in the lungs, leading to fatal heart failure. Current treatments focus on symptom relief but do not address the underlying cause of the disease. However, new therapies like sotatercept, which targets cell proliferation, show promise in reversing the disease. Anti-proliferation drugs are driving a "new wave" of PAH treatments, and their development could also benefit related diseases like pulmonary hypertension secondary to heart failure.

Insights into Brain Cell Material Transfer to Neurons in Mice
neuroscience2 years ago

Insights into Brain Cell Material Transfer to Neurons in Mice

Researchers at UC Davis have discovered how oligodendrocyte-lineage cells transfer cell material to neurons in the mouse brain, providing evidence of a coordinated nuclear interaction between these cells and neurons. This new understanding opens the door for the development of novel therapies for a range of neurodegenerative disorders, such as Alzheimer’s disease, cerebral palsy, Parkinson’s and Huntington’s disease. The study also showed that material transfer to neurons is increased during chronic neuroinflammation, which could lead to better-targeted therapies for conditions caused by the accumulation of pathogenic proteins in neurons.

White House invests $5B to accelerate COVID vaccine development.
health2 years ago

White House invests $5B to accelerate COVID vaccine development.

The Biden administration is launching "Project Next Gen," a $5 billion program to accelerate the development of new coronavirus vaccines and treatments, with a focus on creating long-lasting monoclonal antibodies, accelerating the development of vaccines that produce mucosal immunity, and developing pan-coronavirus vaccines to guard against new variants of SARS-CoV-2 and other coronaviruses. The initiative seeks to better protect against a still-mutating virus, as well as other coronaviruses that might threaten us in the future. The effort comes after Biden health officials had sought for about a year to get the initiative off the ground but were repeatedly rebuffed by Congress.