February 26, 2021February 26, 2021
What ‘Rare’ Means to Me This Rare Disease Day
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
A few days ago, a friend working in healthcare communications asked: “What does rare mean for you?” It got me ... Read more
February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
News, syndicated
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years ... Read more
February 24, 2021February 24, 2021
Umbilical Cord Stem Cells Eased RDEB Symptoms
News
When injected into the bloodstream, mesenchymal stem cells (MSCs) — cells that can grow into different cell types and have ... Read more
February 22, 2021February 22, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
News, syndicated
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature ... Read more
February 18, 2021February 16, 2021
How We Began Educating Our Community About EB
Columns, Fragile but Fierce – a Column by Patrice Williams
There are days when Jonah, my 11-year-old son with epidermolysis bullosa, and I feel like educating folks about his condition. ... Read more
February 17, 2021February 17, 2021
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
News, syndicated
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of ... Read more
February 10, 2021February 11, 2021
Pandemic Won’t Stop Rare Disease Day on Feb. 28
News, syndicated
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities ... Read more
February 4, 2021February 1, 2021
Fighting for Insurance to Cover My Son’s Lifesaving Bandages
Columns, Fragile but Fierce – a Column by Patrice Williams
Jonah, my 11-year-old son who has epidermolysis bullosa (EB), is wounded from head to toe and must be covered in ... Read more
February 3, 2021February 3, 2021
Staph Infections Prevalent in EB Skin Wounds
News
Infection by the bacteria Staphylococcus aureus is detected in most wounds of patients with epidermolysis bullosa (EB), according to an analysis ... Read more
February 2, 2021February 2, 2021
NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
News, syndicated
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State ... Read more
February 2, 2021January 29, 2021
By Focusing on What I Can Control, I’m Finding the Good in 2020
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
This year, I want to say thank you. Thank you, 2020! I know that many will think, “Why ‘thank you’? ... Read more
January 27, 2021January 27, 2021
Phase 3 Trial of EB-101 Cell Therapy Seeks to Complete Enrollment This Year
News
Abeona Therapeutics will proceed with enrollment for its pivotal Phase 3 study of EB-101 as a treatment for recessive dystrophic ... Read more

A few days ago, a friend working in healthcare communications asked: “What does rare mean for you?” It got me thinking. I answered: “Rare is something special, something unique. Something ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

News, syndicated

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 24, 2021February 24, 2021

Umbilical Cord Stem Cells Eased RDEB Symptoms

News

When injected into the bloodstream, mesenchymal stem cells (MSCs) — cells that can grow into different cell types and have potent regeneration properties — can temporarily reduce pain, skin itching ... Read more

February 22, 2021February 22, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

News, syndicated

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more

February 18, 2021February 16, 2021

How We Began Educating Our Community About EB

Columns, Fragile but Fierce – a Column by Patrice Williams

There are days when Jonah, my 11-year-old son with epidermolysis bullosa, and I feel like educating folks about his condition. And there are days when we don’t. When he was ... Read more

February 17, 2021February 17, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 10, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 4, 2021February 1, 2021

Fighting for Insurance to Cover My Son’s Lifesaving Bandages

Columns, Fragile but Fierce – a Column by Patrice Williams

Jonah, my 11-year-old son who has epidermolysis bullosa (EB), is wounded from head to toe and must be covered in lifesaving bandages every single day. These bandages not only cover ... Read more

February 3, 2021February 3, 2021

Staph Infections Prevalent in EB Skin Wounds

News

Infection by the bacteria Staphylococcus aureus is detected in most wounds of patients with epidermolysis bullosa (EB), according to an analysis of a U.S. and Canadian database. The study “Characterization of ... Read more

February 2, 2021February 2, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News, syndicated

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs ... Read more

What ‘Rare’ Means to Me This Rare Disease Day