We thank Michèl Willemsen and colleagues for their interest in the ATTeST trial of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia.1 The two doses studied in ...

The introduction of anti-VEGF agents has revolutionized the field of medical retina care by reducing rates of vision loss and ...

Phase 3 NEAT clinical trial nearing 50% enrollment; company reiterates expectation of enrollment completion in second quarter ...

“We have the first device to treat macular telangiectasia type 2, which has been through the clinical trial program, and the long-term results up to 9 years find that it is still relatively well ...

Quince Therapeutics (QNCX) “announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. Patent Application ...

Martin Friedlander, MD, PhD, discusses phase 3 macular telangiectasia findings, highlighting the potential for a decade-long ...

The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA ...

announced the online publication of safety data from patients with Ataxia-Telangiectasia (A-T) treated with EryDex for a minimum of 24 months in Frontiers in Neurology. "A-T is a rare pediatric ...

GTx-102 is a novel, concentrated oral-mucosal spray of betamethasone intended to improve neurological symptoms of Ataxia-Telangiectasia (A-T), for which there are currently no FDA-approved therapies.

Ophthalmologists must take measures to avoid misdiagnosing age-related macular degeneration and consider mimics of the ...

Quince Therapeutics, Inc. , a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, announced that the U.S. Patent and ...