Rare Disease Patients Face Barriers in Access and Care
An FDA-approved medication can now treat Friedreich's ataxia, but health plan barriers and formulary exclusions keep many patients from receiving it.
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 ...
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Biogen’s Skyclarys is first EU drug for Friedreich’s ataxia
After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU.
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What to know about nervous system disease 'ataxia'
There are many types, including cerebellar ataxia, sensory ataxia, vestibular ataxia, Friedreich’s ataxia and more general forms of hereditary ataxias. The most common symptoms of the disease ...
Design Therapeutics: Not A Complete Washout Despite Setback
Design Therapeutics' GeneTAC platform is revolutionizing genetic disease treatment with small molecules and promising results ...
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 ...
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SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia
for the treatment of Friedreich’s ataxia (FA). Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA.
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) -- (Nasdaq: ...