Rett syndrome study highlights potential for personalized treatments

Though many studies approach the developmental disorder Rett syndrome as a single condition arising from general loss of ...
Pharm Exec

FDA Approves Avlayah for Treatment of Hunter Syndrome

FDA accelerated approval positions Avlayah as the first therapy engineered to cross the blood–brain barrier for MPS II–associated neurologic manifestations, enabling CNS target engagement previously ...
The New England Journal of Medicine

Endovascular Therapy for Post-Thrombotic Syndrome — A Randomized Trial

Post-thrombotic syndrome is common after deep-vein thrombosis and can cause severe symptoms involving the limbs that impair patients’ activity and quality of life. Endovascular therapy can ...
Healio

FDA approves Avlayah to treat neurologic manifestations of Hunter syndrome

Please provide your email address to receive an email when new articles are posted on . Avlayah is delivered to the brain and periphery through Denali Therapeutics’ TransportVehicle Platform. The FDA ...
The BMJ

Treatments for opioid use disorder during pregnancy

Reassuring evidence on neurodevelopmental outcomes Opioid use disorder during pregnancy poses substantial risks of harm to ...
Business Insider

Denali Therapeutics Announces U.S. FDA Approval of AVLAYAH™ (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II)

First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage disease First FDA-approved medicine in emerging new class of biotherapeutics that ...

Global trial shows novel treatment for triple-negative breast cancer nearly doubles survival

A global, multicenter phase III trial, TROPION-Breast02, led by a senior medical oncologist and researcher from the National ...

CNBC Cures: A father and a doctor on how raising a child with Angelman syndrome changed him

A doctor reflects on how raising a son with Angelman syndrome reshaped his family, deepened his empathy and turned grief into ...
AHA/ASA Journals

Targeting Interleukin-8–Mediated Cellular Crosstalk Reverses Hypertrophic Cardiomyopathy and Cardiac Fibrosis in Noonan Syndrome

BACKGROUND: Genetic variants in components or regulators of the RAS-MAPK signaling pathway are causative for severe and early-onset hypertrophic cardiomyopathy (HCM) in patients with Noonan syndrome ...
Oregonian

yao ming

Since the Blazers stunned the NBA universe by acquiring the Chinese center on draft night, he has quickly emerged as one of the most popular — and marketable — players on the roster. The 7-foot-4 ...
eLife

Molidustat Targets a Synthetic Lethal Vulnerability in APC-Mutant Colorectal Cancer through GSTP1 and PHD2 Co-Inhibition

This important paper substantially advances our understanding of how Molidustat may work, beyond its canonical role, by identifying its therapeutic targets in cancer. This study presents a compelling ...
The Lancet

Personalised therapies for children and adolescents with Dravet syndrome

For decades, the treatment of developmental and epileptic encephalopathies has focused on suppressing seizures rather than modifying the underlying biology of disease. In the New England Journal of ...