James Moore – Page 10 – Patient Worthy

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World Orphan Drug Congress USA 2023: Inozyme HCP and Patient Engagement Executive Discuss Importance of Technology and Partnerships in Rare Disease Diagnosis and Treatment

Post author:James Moore
Post published:June 22, 2023
Post category:ABCC6 deficiency ENPP1 Deficiency Generalized arterial calcification of infancy-1 (GACI1)Pseudoxanthoma elasticum

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023, at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…

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Rare Classroom: Antiphospholipid Syndrome

Post author:James Moore
Post published:June 16, 2023
Post category:Antiphospholipid Syndrome

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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World Orphan Drug Congress USA 2023: The Challenge of Ultra-Rare Disease Drug Development

Post author:James Moore
Post published:June 15, 2023
Post category:Barth Syndrome Timely

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…

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Peptide Shows Potential for Treating Eosinophilic Esophagitis in Phase 2 Trial

Post author:James Moore
Post published:June 14, 2023
Post category:Eosinophilic Esophagitis

According to a story from Healio, the pharmaceutical company Revolo Biotherapeutics has been developing '1104, a peptide with a mechanism that 'resets' the immune system to pre-empt the appearance of…

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Rare Classroom: Stevens-Johnson Syndrome

Post author:James Moore
Post published:June 9, 2023
Post category:Stevens-Johnson Syndrome

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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This Combination Treatment Reduced Treatment Failure in Kids with Crohn’s Disease

Post author:James Moore
Post published:June 7, 2023
Post category:Crohn's disease

According to a story from Healio, a recent study found that a treatment combination of adalimumab and low dose methotrexate was able to reduce treatment failure by two times in…

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World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development

Post author:James Moore
Post published:June 7, 2023
Post category:Rare Disease

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…

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Bluebird Bio Submits Application for a Sickle Cell Disease Gene Therapy

Post author:James Moore
Post published:June 6, 2023
Post category:Sickle cell anemia Sickle Cell Disease

According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…

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This Town is Showing Support for a Girl with Batten Disease

Post author:James Moore
Post published:June 5, 2023
Post category:Batten Disease

According to a story from CBS News Boston, Scarlett is just six years old and was recently hit with a devastating diagnosis: Batten disease. Unfortunately, very little can be done…

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Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis

Post author:James Moore
Post published:June 5, 2023
Post category:Rare Disease

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Rare Classroom: Ankylosing Spondylitis

Post author:James Moore
Post published:June 2, 2023
Post category:Ankylosing Spondylitis

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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RDLA Webinar: Mental Health and Rare Diseases

Post author:James Moore
Post published:June 1, 2023
Post category:Rare Disease

On May 18, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…

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Vabysmo Shows Advantages Over Aflibercept in Wet AMD and Diabetic Macular Edema

Post author:James Moore
Post published:May 31, 2023
Post category:Diabetic macular edema Diabetic retinopathy Wet macular degeneration

An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able to dry retinal fluid more…

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May is Prader-Willi Syndrome Awareness Month: Spreading Rare Disease Awareness

Post author:James Moore
Post published:May 26, 2023
Post category:Prader-Willi Syndrome

The month of May is recognized as Prader-Willi Syndrome (PWS) Awareness Month, and May 27th is International PWS Day. This is a time to spread awareness about this rare disease…

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Study of the Week: Hydroxyurea Underused in Pediatric Sickle Cell Anemia Patients Despite Recommendations

Post author:James Moore
Post published:May 22, 2023
Post category:Sickle cell anemia Sickle Cell Disease

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Rare Classroom: Neurofibromatosis Type 2

Post author:James Moore
Post published:May 19, 2023
Post category:Neurofibromatosis Neurofibromatosis Type 2

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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myTomorrows Launches Clinical Trial Tool TrialSearch AI

Post author:James Moore
Post published:May 18, 2023
Post category:Rare Disease

On April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…

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That’s My Word: Spreading Multiple Myeloma Awareness in the Black Community

Post author:James Moore
Post published:May 16, 2023
Post category:Multiple Myeloma

Marsha Calloway-Campbell lives a full life as a lawyer, business owner, wife to her husband Armaray, mother to her three adult daughters, and “Naun” to her toddler grandson. But her…

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Study of the Week: Researchers Identify Racial and Socioeconomic Disparities in Pediatric Bone Cancer Outcomes

Post author:James Moore
Post published:May 8, 2023
Post category:Ewing's family of tumors Osteosarcoma

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Some Pancreatic Cancer Patients Receiving Chemo May Benefit from Antibiotics

Post author:James Moore
Post published:May 5, 2023
Post category:Pancreatic Cancer

According to a story from Healio, a recent study found that patients with metastatic pancreatic cancer (more specifically, pancreatic ductal adenocarcinoma, the most common form) that were being treated with…

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FDA Expands Thyroid Eye Disease Drug Approval Indication

Post author:James Moore
Post published:May 5, 2023
Post category:Thyroid eye disease

According to a story from Healio, the US Food and Drug Administration (FDA) has recently updated the indication for the drug teprotumumab (marketed as Tepezza). Approved for the treatment of…

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Rare Classroom: Stickler Syndrome

Post author:James Moore
Post published:May 5, 2023
Post category:Stickler Syndrome Stickler syndrome- type 3

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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Cystinosis Awareness Day is on May 7: Spreading Rare Disease Awareness

Post author:James Moore
Post published:May 4, 2023
Post category:Cystinosis Cystinosis- ocular nonnephropathic

May 7, 2023 will be recognized as Cystinosis Awareness Day. This will be a day to spread awareness about the rare disease cystinosis among the general public and the medical…

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Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

Post author:James Moore
Post published:May 1, 2023
Post category:Duchenne Muscular Dystrophy

According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…

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Study of the Week: One of Four Atypical Hemolytic Uremic Syndrome Patients Have PTSD

Post author:James Moore
Post published:May 1, 2023
Post category:Atypical hemolytic uremic syndrome

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

World Orphan Drug Congress USA 2023: Inozyme HCP and Patient Engagement Executive Discuss Importance of Technology and Partnerships in Rare Disease Diagnosis and Treatment

Rare Classroom: Antiphospholipid Syndrome

World Orphan Drug Congress USA 2023: The Challenge of Ultra-Rare Disease Drug Development

Peptide Shows Potential for Treating Eosinophilic Esophagitis in Phase 2 Trial

Rare Classroom: Stevens-Johnson Syndrome

This Combination Treatment Reduced Treatment Failure in Kids with Crohn’s Disease

World Orphan Drug Congress USA 2023: The Forefront of Rare Disease Drug Development

Bluebird Bio Submits Application for a Sickle Cell Disease Gene Therapy

This Town is Showing Support for a Girl with Batten Disease

Study of the Week: RNA Sequencing Platform Could Improve Pediatric Rare Disease Diagnosis

Rare Classroom: Ankylosing Spondylitis

RDLA Webinar: Mental Health and Rare Diseases

May is Prader-Willi Syndrome Awareness Month: Spreading Rare Disease Awareness

Study of the Week: Hydroxyurea Underused in Pediatric Sickle Cell Anemia Patients Despite Recommendations

Rare Classroom: Neurofibromatosis Type 2

myTomorrows Launches Clinical Trial Tool TrialSearch AI

That’s My Word: Spreading Multiple Myeloma Awareness in the Black Community

Study of the Week: Researchers Identify Racial and Socioeconomic Disparities in Pediatric Bone Cancer Outcomes

Some Pancreatic Cancer Patients Receiving Chemo May Benefit from Antibiotics

FDA Expands Thyroid Eye Disease Drug Approval Indication

Rare Classroom: Stickler Syndrome

Cystinosis Awareness Day is on May 7: Spreading Rare Disease Awareness

Experimental Duchenne Muscular Dystrophy Treatment Earns Fast Track Designation

Study of the Week: One of Four Atypical Hemolytic Uremic Syndrome Patients Have PTSD

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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