Written by K.M. I have never given much thought to how I would die. Perhaps because my health and longevity has been laid out for me since infancy. Maybe I…
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…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
For the last two and a half years, the COVID-19 pandemic has dominated the headlines. It has been hard to look online – or do anything in person – without…
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…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part Two We were told about what…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part One At this stage, the ED…
This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Oscar was born in January 2020. He was a…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
Maya Doyle is a health social worker specifically focused in pediatrics and the transition from pediatric care to adult care. She has been practicing for twenty years. Now, she works…
Hannah Creel was only 18 months old when she was first diagnosed with cystinosis, a rare disorder that affects all organs in the body, in particular the kidneys. Her parents…
WLNS Brooklyn Michigan is sharing news about Jaxon Meschke, a four-year-old boy with a rare disease called cystinosis. Jaxon’s body has been fighting the disease since he was six…
A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
According to a story from ktvl.com, seven year old Emma Suetta, of Etna, California lives with a rare disease called cystinosis, and she is doing her part to help find…
Written by Rebekah Palmer We have passed the 30th anniversary of the Americans with Disabilities Act. The landmark legislation that prohibits discrimination of disabled people in public spaces is rarely…
The documentary "Walk in My Shoes” takes viewers through a lifetime with cystinosis with Chandler and his family. After Chandler was diagnosed with the rare disease as an infant, making…
The Cystinosis Research Network (CRN) has created a new program to bring light to cystinosis families during these uncertain times. It is called the Brighter Days Care Package Program. Brighter…
My name is Mika Jayne Covington. I’m 29 years old and I live with an ultra-rare disease called cystinosis. I am also a transgender woman and my pronouns are she,…
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…
Because I will be looking for a job from a rare but certainly not unique place of being, I decided to reach out to a mentor who lives with the…
AVROBIO has recently received the Orphan Drug designation for AVR-RD-04, a gene therapy for the treatment of cystinosis. This treatment takes the stem cells of those affected and modifies them…
In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…
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