Rare Disease – Page 59 – Patient Worthy

Oncternal Therapeutics Announces Orphan Drug Designations of Cirmtuzumab ROR1 Antibody for Treatment of Mantle Cell Lymphoma and for Treatment of Chronic Lymphocytic Leukemia

Post author:Rose Duesterwald
Post published:June 30, 2020
Post category:Rare Disease

Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that the United States Food and Drug Administration (FDA) has granted…

Scientists Find New Pathway Leading to Less Inflammation in Asthma and Other Allergic Diseases

Post author:Rose Duesterwald
Post published:June 30, 2020
Post category:Rare Disease

Why are most people able to tolerate cat’s hair while some people have severe reactions to the ubiquitous and practically invisible dust mites? A recent SciTech Daily article followed La…

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FDA Approves CRYSVITA for Tumor-Induced Osteomalacia

Post author:Kendall Mason
Post published:June 29, 2020
Post category:Rare Disease

Ultragenyx Pharmaceutical's therapy, CRYSVITA, has recently been approved by the FDA for the treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors. This approval comes after…

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Enrollment for Phase 2 Cystic Fibrosis Trial to Resume

Post author:James Moore
Post published:June 29, 2020
Post category:Cystic Fibrosis Rare Disease

According to a story from Cystic Fibrosis News Today, Eloxx Pharmaceuticals had just announced recently that it is officially re-opening enrollment for its phase 2 clinical study. This trial will…

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Psoriasis Drug Now a Potential Candidate to Treat PsA and Uveitis

Post author:Jessica Lynn
Post published:June 29, 2020
Post category:Psoriatic Arthritis Rare Disease Uveitis

According to LabioTech, a psoriasis drug developed by biotechnology company Affibody showed promising results in a Phase II clinical trial (AFFIRM-35). This trial, which consisted of 108 participants with…

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ANCA Vasculitis Patients at an Increased Risk of Depression, Study Finds

Post author:James Moore
Post published:June 26, 2020
Post category:Granulomatosis with Polyangiitis Rare Disease Vasculitis

According to a story from ANCA Vasculitis News, a recent review study has determined that patients living with ANCA vasculitis and other related diseases are at a higher than average…

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Data Suggests MicroRNA-483 Could Ease PAH Symptoms

Post author:Jessica Lynn
Post published:June 26, 2020
Post category:pulmonary arterial hypertension Rare Disease

For most drug therapies, researchers test on animal models of a disease before testing on humans. Recently, using microRNA-483, a small RNA molecule, on rat models of PAH reduced…

Editor’s Choice: New Discoveries and Positive Results

Post author:Colby Rogers
Post published:June 26, 2020
Post category:Chronic urticarias COVID-19 Rare Disease TRAF7 Syndrome

Happy Friday! The world could always use more good news, and this week we've got some new discoveries to discuss. Up first, details on a trial that may have finally…

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Alexion Donates $1.1M to Rare Disease Community

Post author:Jessica Lynn
Post published:June 25, 2020
Post category:COVID-19 Rare Disease

As you know, COVID-19 has fundamentally changed our daily life. What began as a novel coronavirus in Wuhan, China has developed into a global pandemic: 9 million diagnosed cases…

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NAFLD and NASH Predicted to Be a Growing Health Emergency

Post author:Jessica Lynn
Post published:June 25, 2020
Post category:Nonalcoholic steatohepatitis Rare Disease

In a Business Wire press release, the Canadian NASH Network (CanNASH) shared the results of a recent study predicting a severe increase in cases of nonalcoholic fatty liver disease…

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International Panel Gives Expert Advice on the Management of COVID-19 and Blood Cancers

Post author:Rose Duesterwald
Post published:June 25, 2020
Post category:Acute Myeloid Leukemia Myelodysplastic syndromes Rare Disease

COVID-19 has now taken hold in over one hundred eighty countries. According to a recent article in The Lancet, in May of 2020, the United States reported the highest…

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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

Post author:Sunniva Bean
Post published:June 24, 2020
Post category:Cystinosis Fabry Disease Rare Disease

As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…

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Rare Diseases Clinical Research Network Launches COVID-19 Survey in Spanish

Post author:James Moore
Post published:June 24, 2020
Post category:COVID-19 Rare Disease

The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…

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Cures Within Reach Announces the Winner of CureAccelerator Live! for Rare Diseases

Post author:Kendall Mason
Post published:June 24, 2020
Post category:Rare Disease

Cures Within Reach is an organization that aims to help patients by repurposing research. They work towards getting treatments quickly and safely in an effort to aid those who need…

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An Experimental Treatment for Primary Hyperoxaluria has Earned Rare Pediatric Disease Designation

Post author:James Moore
Post published:June 24, 2020
Post category:Chronic Kidney Disease Kidney Stones Primary Hyperoxaluria Type 1 Primary hyperoxaluria type 2 Rare Disease

According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to…

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Dr. Eunice Wang Discusses Newly Approved Treatments for Acute Myeloid Leukemia

Post author:Rose Duesterwald
Post published:June 24, 2020
Post category:Acute lymphoblastic leukemia Acute Myeloid Leukemia Chronic Lymphocytic Leukemia Rare Disease

Earlier this year, The Leukemia & Lymphoma Society hosted a presentation by Dr. Eunice Wang, Clinical Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center. Dr. Wang’s presentation…

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June 24: Celebrate “Mobility Day” with Variety

Post author:Jessica Lynn
Post published:June 24, 2020
Post category:Nemaline Myopathy Rare Disease

If you're like me, your life has changed immensely over the past few months. COVID-19, a novel coronavirus with 8.39 million diagnoses worldwide, has kept us inside and changed…

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Webinar: The Impact of COVID-19 on Medical Discovery

Post author:James Moore
Post published:June 23, 2020
Post category:COVID-19 Rare Disease Timely

Last week, I had the privilege of attending a webinar organized by Research!America titled Partnership, Progress, Pandemic: The Impact of COVID-19 on Medical Discovery. As implied in the title, the…

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New Treatment Approved in Canada for Gastrointestinal Stromal Tumor

Post author:James Moore
Post published:June 23, 2020
Post category:Gastrointestinal Stromal Tumors Rare Disease Timely

According to a story from Biotech 365, the biopharmaceutical company Deciphera Pharmaceuticals has recently announced that the company's drug ripretinib (marketed as QINLOCK™) has been approved by Health Canada. The…

Perturbations in the Body’s Internal Clock Linked to Greater Risk of Parkinson’s, Study Finds

Post author:Rose Duesterwald
Post published:June 23, 2020
Post category:Rare Disease

Older men who have a weak or irregular circadian rhythm — the body’s internal clock that controls the sleep-wake cycle — are approximately three-times more likely to develop Parkinson’s disease…

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New Investigative Therapy for Acute Myeloid Leukemia and Myelodysplastic Syndromes is Being Investigated

Post author:Trudy Horsting
Post published:June 23, 2020
Post category:Acute Myeloid Leukemia Myelodysplastic syndromes Rare Disease

Antigen-specific T-cell therapy is an innovative treatment option which uses immune cells to combat cancer cells. AdventHealth is the very first in the world to use this treatment for patients…

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CAAR T-Cell Therapy as a Treatment for Myasthenia Gravis

Post author:Kendall Mason
Post published:June 22, 2020
Post category:Myasthenia Gravis Rare Disease

According to Myasthenia Gravis News, CAAR T-cell therapy may be a viable treatment option for those with myasthenia gravis. Researchers from the University of Pennsylvania, Yale Medical School, and Cabaletta…

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CDC Hosts Webinar on the Struggles of Those with Chronic Diseases During COVID-19 crisis

Post author:Octavia Walker
Post published:June 22, 2020
Post category:COVID-19 Rare Disease

During this time of COVID-19, many people have some general information about COvID-19 including the knowledge that individuals over 65 years of age are at higher risk of contracting…

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Study Finds Success in Combination Treatment for AL Amyloidosis

Post author:James Moore
Post published:June 22, 2020
Post category:Amyloidosis Rare Disease

According to a story from ajmc.com, a recent study found that a new combination treatment for systemic amyloid light-chain (AL) amyloidosis is more effective than the current standard treatment. The…

Regeneron Pharmaceuticals is Racing to Have an Antibody “Cocktail” Against COVID-19

Post author:Rose Duesterwald
Post published:June 20, 2020
Post category:COVID-19 Rare Disease

Regeneron Pharmaceuticals recently announced that it is testing a medicine for the treatment and/or prevention of Covid-19. According to an article by CNN, the testing of its anti-SARS-CoV-2 antibody combination…

Oncternal Therapeutics Announces Orphan Drug Designations of Cirmtuzumab ROR1 Antibody for Treatment of Mantle Cell Lymphoma and for Treatment of Chronic Lymphocytic Leukemia

Scientists Find New Pathway Leading to Less Inflammation in Asthma and Other Allergic Diseases

FDA Approves CRYSVITA for Tumor-Induced Osteomalacia

Enrollment for Phase 2 Cystic Fibrosis Trial to Resume

Psoriasis Drug Now a Potential Candidate to Treat PsA and Uveitis

ANCA Vasculitis Patients at an Increased Risk of Depression, Study Finds

Data Suggests MicroRNA-483 Could Ease PAH Symptoms

Editor’s Choice: New Discoveries and Positive Results

Alexion Donates $1.1M to Rare Disease Community

NAFLD and NASH Predicted to Be a Growing Health Emergency

International Panel Gives Expert Advice on the Management of COVID-19 and Blood Cancers

Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

Rare Diseases Clinical Research Network Launches COVID-19 Survey in Spanish

Cures Within Reach Announces the Winner of CureAccelerator Live! for Rare Diseases

An Experimental Treatment for Primary Hyperoxaluria has Earned Rare Pediatric Disease Designation

Dr. Eunice Wang Discusses Newly Approved Treatments for Acute Myeloid Leukemia

June 24: Celebrate “Mobility Day” with Variety

Webinar: The Impact of COVID-19 on Medical Discovery

New Treatment Approved in Canada for Gastrointestinal Stromal Tumor

Perturbations in the Body’s Internal Clock Linked to Greater Risk of Parkinson’s, Study Finds

New Investigative Therapy for Acute Myeloid Leukemia and Myelodysplastic Syndromes is Being Investigated

CAAR T-Cell Therapy as a Treatment for Myasthenia Gravis

CDC Hosts Webinar on the Struggles of Those with Chronic Diseases During COVID-19 crisis

Study Finds Success in Combination Treatment for AL Amyloidosis

Regeneron Pharmaceuticals is Racing to Have an Antibody “Cocktail” Against COVID-19

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