Rare Classroom: Williams 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
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“Save Our Medicine:” NPC Families Fight to Save Experimental Medication Access (Pt. 2)
- December 1, 2021
Study for Vision Loss from NF1 Optic Pathway Glioma Funded By Gilbert Family Foundation
Remembering Rachael Kaflowitz: One Mother’s Fight to Support NPC Research and Awareness (Pt. 3)
RGX-202 for DMD Earns Orphan Drug Designation
PODCAST: WAIT, HOW DO YOU SPELL THAT?
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Rare Classroom: Acalvaria
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
Science Simplified: What is Gene Therapy?
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
Study of the Week: The High Cost Burden of Rare Diseases
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
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Chronic Lymphocytic Leukemia
Study Shows Novel BTK Degrader Has Meaningful Results for Patients with Chronic Lymphocytic Leukemia
According to a recent article, a study looking at an investigational BTK degrader proved to have important results in patients with chronic lymphocytic leukemia. Chronic
porphyria
Givlaari Access for Porphyria Expected in February 2022 in England
In late October 2021, the National Institute for Care and Excellence (NICE) in England recommended Givlaari (givosiran) to treat patients with acute porphyria — or,
Ocular Melanoma
Fundraiser Supports Father with Ocular Melanoma
In 2019, Mike Smith, a father of two, was initially diagnosed with ocular melanoma in his right eye. Shortly after, Mike made the necessary –
Rare Disease
Celebrating National Family Caregivers Month
November is officially recognized as National Family Caregivers Month, a time dedicated to recognizing and celebrating caregivers. The entire month is full of recognizing the
Bardet-Biedl Syndrome
Study Looks at Sleep Patterns in Adult Patients with Bardet-Biedl Syndrome
According to a recent article, a study looked at the symptoms of Bardet-Biedl syndrome that can affect a patient’s sleep patterns. Bardet-Biedl Syndrome Bardet-Biedl syndrome
Hunter Syndrome
FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome.
Klippel Trenaunay syndrome
Golfer Casey Martin Has His Leg Amputated
Casey Martin is well known as the golfer who sued the PGA in 2001 and won a landmark 7-2 Supreme Court decision. According to a
Rare Disease
FDA Grants Fasenra Orphan Drug and Fast Track Designations for EG and EGE
The FDA grants certain designations, such as the Orphan Drug (ODD) and Fast Track designations (FTD), to medications intended to treat rare and severe disorders.
Acromegaly
Phase 2 Data Shows Cimdelirsen Benefits for Acromegaly
According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals (“Ionis”), topline data from a Phase 2 clinical trial highlighted the benefits
Ehlers Danlos Syndrome
How Lina Williamson Works to Empower the Ehlers-Danlos Syndrome Community
“The patient network is incredibly important,” Lina Williamson, PhD, explained to me. You see, in her childhood, Lina experienced a number of seemingly unexplainable symptoms:
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