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 ...

Announced Phase 3 STRIVE-ON Safety Trial Met Primary Endpoint and Provided Clinical Benefit Compared to Orally Administered Nimodipine; New Drug Application (NDA) Submission Anticipated First Half of ...

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 ... GTx-104’s potential to ...

Participants will be randomized (1:1) between EryDex or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the ...

Ataxia-Telangiectasia (A-T), today, Friday, February 7, 2025, beginning at 10:00 a.m. Eastern Time. The webinar will feature leading A-T KOL Mary Kay Koenig, M.D., who will: 1) provide an A-T natural ...

Can be done at home. Cons: Costly. Must be used indefinitely. Evidence of efficacy is mixed. Cost: For at-home treatment, a one-time cost of $500 to $3,000 for the apparatus. What it is: A ...

The treatment was generally well-tolerated, with most adverse events being mild to moderate. This recent data supports further evaluation of EryDex for Ataxia-Telangiectasia treatment. These ...

SOUTH SAN FRANCISCO, Calif. - Quince Therapeutics, Inc. (NASDAQ: QNCX), a biotech firm focusing on rare diseases with a current market capitalization of $67.76 million, has received a Notice of ...

Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s ...

The treatment was generally well-tolerated, with most adverse events being mild to moderate. This recent data supports further evaluation of EryDex for Ataxia-Telangiectasia treatment. These ...

By Zach Montague Reporting from Washington President Trump signed an executive order on Tuesday taking steps to end gender-affirming medical treatments for children and teenagers under 19 ...