News

August 19, 2019

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

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

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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, 2019August 9, 2019

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

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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

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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

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

July 26, 2019

Gene-modified Skin Cells Show Promise for RDEB in Phase 1 Trial

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An experimental cell therapy where a patient’s own skin cells (fibroblasts) are engineered in the lab to carry a healthy COL7A1 gene and then injected back into the patient ... Read more

July 19, 2019

Transplants Using Patients’ Own Skin May Be Safe, Effective Treatment for RDEB, Trial Suggests

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Transplanting a patient’s own healthy skin to treat their skin ulcers was found to be safe and effective for three people with recessive dystrophic epidermolysis bullosa (RDEB) for more ... Read more

July 18, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

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A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of ... Read more

July 12, 2019July 17, 2019

FDA Releases Guidelines to Push Industry Development of New Therapies

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The U.S. Food and Drug Administration (FDA) has released a guidance document to assist and encourage industry in developing new treatment options for the rare skin disorder epidermolysis bullosa ... Read more

July 9, 2019

Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients

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The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the ... Read more

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