Cindy Howry: FDA Approves First Treatment for Ataxia-Telangiectasia
Cindy Howry, Executive Principal at Howry Solutions, shared a post on LinkedIn:
”FDA Approves First Treatment for Ataxia-Telangiectasia
The FDA has approved Aqneursa (levacetylleucine), an oral suspension that marks the first-ever treatment specifically approved for ataxia—a progressive neurological condition that affects movement and coordination—in patients with ataxia-telangiectasia.
The approval applies to adults and children weighing at least 15 kg (about 33 pounds).
This represents a significant milestone for a rare genetic disorder that has had no targeted treatment options until now.
Aqneursa builds on the medication’s prior FDA approval in 2024 for treating neurological symptoms in another rare genetic condition, Niemann-Pick disease type C.”
Stay updated with Hemostasis Today.
-
Nov 8, 2026, 12:22Marc Carrier: Advancing Catheter-Related Thrombosis Prevention Through Factor XI Inhibition
-
Nov 8, 2026, 11:56Why Genetic Predisposition Does Not Always Lead to Thrombosis – Stork Genetics
-
Nov 8, 2026, 11:45Sher-Lu Pai: Investigating Intracardiac Thrombosis and Its Management in Liver Transplantation
-
Nov 8, 2026, 11:25Mehmet Cilingiroglu: Navigating Anticoagulation Choices in Acute Coronary Syndrome PCI
-
Nov 8, 2026, 07:16Gilberto de lima Lopes Junior: The Cardiovascular Consequences of Cancer in Younger Patients
-
Nov 8, 2026, 06:51Haney Mallemat: Key Principles and Pitfalls of LVAD Emergencies
-
Nov 8, 2026, 06:31Maria Omer: Clinical Recognition and Management of Cavernous Sinus Thrombosis
-
Nov 8, 2026, 06:16Natasha Jolakoski: University of New Mexico Hospital Promotes PGY-2 Thrombosis and Hemostasis Management Pharmacy Residency
-
Nov 8, 2026, 05:47Safaa Hilal: POCUS in the Evaluation of Perioperative Troponin Elevation