Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure
Quinton's Quest for a Cure (photo submitted by Quinton's mother, Laura Nitahara)

Raising Money for Krabbe Disease Research: Quinton’s Quest for a Cure

When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…

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Beta Thalassemia Major: This Couple Had a Baby to Save Their Son
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Beta Thalassemia Major: This Couple Had a Baby to Save Their Son

According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…

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Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation
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Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation

According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…

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UT Southwestern Expands Diagnostic and Treatment Approaches for Aplastic Anemia, MDS
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UT Southwestern Expands Diagnostic and Treatment Approaches for Aplastic Anemia, MDS

What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…

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Experimental Gene Therapy for GM1 Gangliosidosis Earns Rare Pediatric Disease Designation
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Experimental Gene Therapy for GM1 Gangliosidosis Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…

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IB1001 Effective for NPC Treatment, Study Says
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IB1001 Effective for NPC Treatment, Study Says

According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…

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First Patient Dosed in Trial of Hypoparathyroidism Treatment, AZP-3601
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First Patient Dosed in Trial of Hypoparathyroidism Treatment, AZP-3601

Amolyt Pharma has recently announced that they have dosed the first patient in their trial of AZP-3601, a treatment for hypoparathyroidism. According to GlobeNewswire, this treatment will adequately address the…

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FDA Grants IDE to Test Experimental Device for Pulmonary Arterial Hypertension
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FDA Grants IDE to Test Experimental Device for Pulmonary Arterial Hypertension

According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…

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The FDA Has Provided 16 Million Dollars to Scientists to Continue Rare Disease Research During the Pandemic
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The FDA Has Provided 16 Million Dollars to Scientists to Continue Rare Disease Research During the Pandemic

 The United States FDA has just announced that they will be providing  6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…

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New Collaboration: Scribe Therapeutics and Biogen to Explore CRISPR-Related Therapies for ALS

On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…

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Two Epidermolysis Bullosa Nonprofits United to Make the Largest Global Organization for EB

EB Research Partnership (EBRP) is based out of New York, United States. EB Research Foundation (EBRF) is an organization based out of Australia with the same goals. The two organizations  have…

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Comic Book Artist J.G. Jones on His Project Illustrating Patients with Rare Blood Cancers

J.G. Jones is a renowned comic book artist, illustrating covers of beloved superhero novels like Batman and Robin, Doc Savage, and Frankenstein. He has probably worked for all of the…

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Trodelvy Gets Orphan Drug Designation as a Glioblastoma Treatment
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Trodelvy Gets Orphan Drug Designation as a Glioblastoma Treatment

According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…

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30 New Genes Affiliated with Charcot Marie Tooth Disease Identified

Researchers have just uncovered 30 new genes that may cause Charcot-Marie-Tooth Disease (CMT).  These genes were uncovered using computer analyses of biological data, a process called bioinformatics. This study was published…

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Phase 3 Study Results Bring Hope for Children with Primary Hyperoxaluria Type 1  
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Phase 3 Study Results Bring Hope for Children with Primary Hyperoxaluria Type 1  

  According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…

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The Glanzmann’s Research Foundation: Dedicated to Finding a Cure
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The Glanzmann’s Research Foundation: Dedicated to Finding a Cure

Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…

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