FDA Removes Clinical Hold on Nomlabofusp Study for Friedreich’s Ataxia
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FDA Removes Clinical Hold on Nomlabofusp Study for Friedreich’s Ataxia

Three years after the U.S. Food and Drug Administration (FDA) first placed a full clinical hold on Larimar Therapeutics’ Phase 2 clinical study, the hold has officially been lifted. The…

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ClimbFARACure: How Summiting Aconcagua Raised Friedreich’s Ataxia Awareness
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ClimbFARACure: How Summiting Aconcagua Raised Friedreich’s Ataxia Awareness

It was never just about reaching the summit of Aconcagua, the highest mountain in both the Western and Southern hemispheres. For Scott Osleeb, scaling the heights of this remarkable peak…

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Rare Community Profiles: From Afraid to Advocate: How Natalie Found Her Power After a Friedreich’s Ataxia Diagnosis
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Rare Community Profiles: From Afraid to Advocate: How Natalie Found Her Power After a Friedreich’s Ataxia Diagnosis

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

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Now Approved: Skyclarys for Friedreich Ataxia
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Now Approved: Skyclarys for Friedreich Ataxia

  Until recently, the only therapeutic options for people with Friedreich ataxia were symptomatic and focused on symptom management: mobility aids, surgery, psychotherapy, speech therapy, heart medications, hearing and vision…

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Potential Treatment for Friedreich’s Ataxia Earns Orphan Drug and Rare Pediatric Disease Designations
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Potential Treatment for Friedreich’s Ataxia Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…

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RT001 Granted Rare Pediatric Disease Designation for INAD, FA
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RT001 Granted Rare Pediatric Disease Designation for INAD, FA

In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…

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Australian Patent Issued for Trehalose (SLS-005) for Neurodegenerative Disorders
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Australian Patent Issued for Trehalose (SLS-005) for Neurodegenerative Disorders

In a press release from late January 2021, biopharmaceutical company Seelos Therapeutics, Inc. ("Seelos") announced the issuance of an Australian patent for Trehalose (SLS-005). The patent (#2019204513) centers around the…

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The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy
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The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy

According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…

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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial
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ICYMI: First Patient is Dosed in Phase 2 Friedreich’s Ataxia Trial

According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…

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Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries

The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…

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Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?

The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…

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Electrical Brain Stimulation Could Effectively Treat Neurodegenerative Ataxias

According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…

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New Expanded Access Trials For FENIB and LOTS Patients Set to Begin
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New Expanded Access Trials For FENIB and LOTS Patients Set to Begin

According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…

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