News

June 25, 2019

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

News

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials ... Read more

June 21, 2019

Vital Signs Monitor May Lower Risk of Skin Injury in NICU Babies and EB Patients

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A skin-like device that measures heart rate, breathing, blood oxygen, blood pressure, and body temperature may provide a safer way to monitor vital signs of those in intensive care ... Read more

June 20, 2019

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

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Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, ... Read more

June 19, 2019June 17, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most ... Read more

June 14, 2019June 18, 2019

EB-101, Potential Cell Therapy for RDEB, Seen to Lead to Sustained Wound Healing in Trial

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EB-101, a cell therapy candidate from Abeona Therapeutics, induced sustained wound healing lasting for three years or more in a large proportion of people with recessive dystrophic epidermolysis bullosa (RDEB), ... Read more

June 7, 2019

RDEB Gene Therapy Candidate FCX-007 Given FDA Regenerative Medicine Advanced Therapy Status

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Fibrocell Science’s investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) has received the regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and ... Read more

May 31, 2019May 31, 2019

First Patient Dosed in Phase 2 Trial Testing RGN-137 for Epidermolysis Bulllosa

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The first patient with epidermolysis bullosa has received treatment with RegeneRx Biopharmaceuticals‘ investigational therapy RGN-137 in a Phase 2 clinical trial in the U.S., the company announced. RGN-137 is a ... Read more

May 27, 2019May 24, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

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RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site ... Read more

May 24, 2019

Gene Therapy Using CRISPR-Cas9 Shows Promise as Safer, More Efficient Method to Treat RDEB, Preliminary Study Shows

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Gene therapy using CRISPR-Cas9 enabled precise correction in the lab of defective skin cells from a patient with recessive dystrophic epidermolysis bullosa, showing promise as a safer and more ... Read more

May 17, 2019

ProQR Creates New Company, Wings Therapeutics, to Assume Development of QR-313 for Dystrophic EB

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A new company called Wings Therapeutics has been tasked with continuing the development of QR-313, an investigational RNA therapy for dystrophic epidermolysis bullosa (DEB), following a spin-out from ProQR ... Read more

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