September 13, 2019
DAAC Dressing Closes EB Wounds Faster Than Standard Saline-Antibiotic One, Study Says
News
A type of wound dressing, called dialkylcarbamoylchloride (DAAC)‐coated cotton acetate, closes infected wounds related to epidermolysis bullosa (EB) faster ... Read more
September 6, 2019
Cell Therapy for RDEB Needs Collagen VII Repair in Two Types Skin Cells to Achieve Stability, Study Finds
News
Cell therapy with genetically engineered skin grafts requires that collagen type VII be “corrected” in two types of skin ... Read more
September 2, 2019August 30, 2019
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
News
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as ... Read more
August 30, 2019
First Patient Dosed in Phase 2 Trial of RGN-137 Achieves Complete Wound Healing, RegeneRx Announces
News
RegeneRx Biopharmaceuticals announced the first patient enrolled in a Phase 2 clinical trial of RGN-137, the company’s investigational topical ... Read more
August 29, 2019August 28, 2019
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
News
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert ... Read more
August 23, 2019
Injecting Engineered Cells May Prevent Blistering in RDEB, Study Indicates
News
Injection of mesenchymal stromal cells (MSCs) — cells that can be isolated from several tissues and may grow into ... Read more
August 19, 2019
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
News
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you ... Read more
August 16, 2019
Amryt Releases Design of Pivotal Trial Evaluating Oleogel-S10 for EB Patients of All Ages
News
Amryt Pharma is conducting a Phase 3 clinical trial evaluating the effectiveness and long-term safety of Oleogel-S10 (AP101), a ... Read more
August 9, 2019September 10, 2019
Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients
News
With its lead therapy candidate AGLE-103 set to begin clinical testing in the coming months, Aegle Therapeutics is being honored with ... Read more
August 2, 2019August 1, 2019
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
News
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks ... Read more
August 1, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
News
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has ... Read more
July 26, 2019
Gene-modified Skin Cells Show Promise for RDEB in Phase 1 Trial
News
An experimental cell therapy where a patient’s own skin cells (fibroblasts) are engineered in the lab to carry a ... Read more

DAAC Dressing Closes EB Wounds Faster Than Standard Saline-Antibiotic One, Study Says

A type of wound dressing, called dialkylcarbamoylchloride (DAAC)‐coated cotton acetate, closes infected wounds related to epidermolysis bullosa (EB) faster than the standard saline dressing plus a topic antibiotic, and ... Read more

September 6, 2019

Cell Therapy for RDEB Needs Collagen VII Repair in Two Types Skin Cells to Achieve Stability, Study Finds

News

Cell therapy with genetically engineered skin grafts requires that collagen type VII be “corrected” in two types of skin cells — keratinocytes and fibroblasts — to be effective against ... Read more

September 2, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 30, 2019

First Patient Dosed in Phase 2 Trial of RGN-137 Achieves Complete Wound Healing, RegeneRx Announces

News

RegeneRx Biopharmaceuticals announced the first patient enrolled in a Phase 2 clinical trial of RGN-137, the company’s investigational topical gel for treating epidermolysis bullosa (EB), achieved complete wound healing. RGN-137 ... Read more

August 29, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 23, 2019

Injecting Engineered Cells May Prevent Blistering in RDEB, Study Indicates

News

Injection of mesenchymal stromal cells (MSCs) — cells that can be isolated from several tissues and may grow into different cell types when cultured in a lab dish — ... Read more

August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 16, 2019

Amryt Releases Design of Pivotal Trial Evaluating Oleogel-S10 for EB Patients of All Ages

News

Amryt Pharma is conducting a Phase 3 clinical trial evaluating the effectiveness and long-term safety of Oleogel-S10 (AP101), a topical formulation of birch bark extract, for accelerating wound healing ... Read more

August 9, 2019September 10, 2019

Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients

News

With its lead therapy candidate AGLE-103 set to begin clinical testing in the coming months, Aegle Therapeutics is being honored with a Partners in Progress Award by the Dystrophic Epidermolysis Bullosa ... Read more

August 2, 2019August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more