Design Therapeutics: Not A Complete Washout Despite Setback
Design Therapeutics' GeneTAC platform is revolutionizing genetic disease treatment with small molecules and promising results ...
Larimar started at buy by Truist on Friedreich's ataxia drug
Truist has initiated coverage of Larimar (LRMR) with a buy rating, citing an "asymmetric" risk/reward ahead of regulatory ...
Optimistic Outlook on Larimar Therapeutics’ Nomlabofusp: Advancements in Clinical Trials for Adolescents with Friedreich’s Ataxia
Leerink Partners analyst Joseph Schwartz has reiterated their bullish stance on LRMR stock, giving a Buy rating on January 24.Invest with ...
Quince Therapeutics Announces Frontiers in Neurology Publication of Long-Term Safety of EryDex in Pediatric Patients with Ataxia-Telangiectasia
Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s ...
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation ...
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What to know about nervous system disease 'ataxia'
In such cases, an inability to balance is a symptom known as ataxia. But ataxia can also be its own condition or disease. Start the day smarter. Get all the news you need in your inbox each morning.
New type of cerebellar ataxia discovered: How the immune system destroys the cerebellum
Cerebellar ataxia is a neurological disorder of the cerebellum. This important area at the back of the brain acts as a ...
Solid Biosciences' Ataxia Treatment Receives FDA Fast-Track Designation
Fast-track status will allow Solid Biosciences to have more frequent meetings with the FDA throughout the treatment-development process and, potentially, get the product to patients faster than would ...
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Ataxia Patient MRI (IMAGE)
The ataxia patient's MRI shows cerebellar atrophy and cervical spinal cord thinning. Repeat MRIs showed the atrophy slowly progressed over time. Advanced Search ...
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Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE ...