Encouraging Results from a Phase 2 Study of IL-2 to Treat Alzheimer’s Patients
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Encouraging Results from a Phase 2 Study of IL-2 to Treat Alzheimer’s Patients

BioSpace recently published a press release discussing the interim results highlighting a Coya Therapeutics’ study. The study evaluates the safety, tolerability, and biological activity of LD IL-2 in 38 patients…

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New Podcast Episode: 50 Years of Supporting the TSC Community, feat. TSC Alliance President and CEO Kari Rosbeck
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New Podcast Episode: 50 Years of Supporting the TSC Community, feat. TSC Alliance President and CEO Kari Rosbeck

Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kari Rosbeck, the…

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Enrollment Completed in Clinical Trials for Primary Sjögren’s Syndrome and IgA Nephropathy
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Enrollment Completed in Clinical Trials for Primary Sjögren’s Syndrome and IgA Nephropathy

Jianmin Fang, M.D. the CEO of the biotechnology company RemeGen, recently announced in PRNewswire that the company is continuing its significant progress in its research of Telitacicept (RC18) to treat…

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First Patient Enrolled in VE303 Study for Recurrent C. Diff
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First Patient Enrolled in VE303 Study for Recurrent C. Diff

An estimated 25% of people with Clostridioides difficile (C. diff) infections experience recurrence following successful antibiotic intervention. After three or more recurrent infections, the risk of developing another infection sits…

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AGMB-447 Nabs Orphan Drug Designation as Potential IPF Treatment
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AGMB-447 Nabs Orphan Drug Designation as Potential IPF Treatment

People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics ("Agomab") is working to introduce another potential therapeutic…

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Congressional Briefing Highlights Impact of Research Funding on Rare Eye Diseases: Retinitis Pigmentosa and Stargardt Disease
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Congressional Briefing Highlights Impact of Research Funding on Rare Eye Diseases: Retinitis Pigmentosa and Stargardt Disease

Written by Judith Hill, Communications and Events Director, National Alliance for Eye and Vision Research In a remarkable show of unity and commitment to eye health, the Alliance for Eye…

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UC Davis Health Researchers Identify Biomarkers for Schizophrenia in 22q11.2 Deletion Syndrome

A 2008 report published in Current Psychiatry Reports found that approximately 1% of people with schizophrenia also have a rare genetic disorder called 22q11.2 deletion syndrome. This suggests that schizophrenia…

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“An Event to End NF” Helped Teen Raise Funds, Awareness for Neurofibromatosis
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“An Event to End NF” Helped Teen Raise Funds, Awareness for Neurofibromatosis

On May 17, 2024, Ohio was graced with a unique, Wizard of Oz-themed fundraising event called "An Event to End NF." NF, in this case, stands for neurofibromatosis, a rare genetic…

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Phase 3 Results: Imsidolimab Provides Positive Outlook in Generalized Pustular Psoriasis (GPP)
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Phase 3 Results: Imsidolimab Provides Positive Outlook in Generalized Pustular Psoriasis (GPP)

Just about one month ago, clinical-stage biotechnology company AnaptysBio announced the availability of positive top-line results from two Phase 3 clinical studies: GEMINI-1 and GEMINI-2. Both studies sought to assess…

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Women More Likely to Develop De Novo Conditions Within One Year of Cushing Syndrome Remission
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Women More Likely to Develop De Novo Conditions Within One Year of Cushing Syndrome Remission

Researchers recently linked Cushing syndrome remission to the development of de novo (first occurrence) diseases, including autoimmune diseases, in various patients. But how did they reach this point? The first…

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ICYMI: Make HStory Platform Offers Hidradenitis Suppurativa Education and Community Support

The importance of physician education and community support in healthcare cannot be understated. Well-educated physicians are better equipped to diagnose, treat, and manage a range of medical conditions to ensure…

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Suicidal Bipolar Depression: Clinical Trial Results of NRX-101 Found Superior Safety and Similar Efficacy Compared to Lurasidone
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Suicidal Bipolar Depression: Clinical Trial Results of NRX-101 Found Superior Safety and Similar Efficacy Compared to Lurasidone

People who have bipolar depression face a 50% lifetime risk of attempting suicide and a 20% risk of death from suicide. Over seven million people in the U.S. are struggling…

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Rare Community Profiles: #RAREis: Increasing Medical Access and Equity for the Hispanic Community through The Akari Foundation’s Initiatives
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Rare Community Profiles: #RAREis: Increasing Medical Access and Equity for the Hispanic Community through The Akari Foundation’s Initiatives

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

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The Dystonia Diagnosis and Prognosis
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The Dystonia Diagnosis and Prognosis

“You have dystonia” are three words that can be very confusing and frightening. Receiving the news that you have any chronic condition can bring out a lot of emotions. People…

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AbbVie Releases Positive Topline Results from Upadacitinib Study for Giant Cell Arteritis

RINVOQ (upadacitinib) is a Janus Kinase inhibitor that is currently approved to treat a variety of conditions: moderate to severe atopic dermatitis, active psoriatis arthritis, moderate to severe rheumatoid arthritis,…

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Rare Community Profiles: How Her Daughter’s MCTD Led Ronda to Help Families Navigate Rare Disease Diagnoses
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Rare Community Profiles: How Her Daughter’s MCTD Led Ronda to Help Families Navigate Rare Disease Diagnoses

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

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Investigational New Drug (IND) Application Accepted for EB-105 to Treat Diabetic Macular Edema
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Investigational New Drug (IND) Application Accepted for EB-105 to Treat Diabetic Macular Edema

An Investigational New Drug (IND) application is a crucial part of the drug development process. INDs are requests submitted to the U.S. Food and Drug Administration (FDA) that authorize the…

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