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Latest Genodermatoses Stories

2014-04-01 08:28:31

DURHAM, N.C., April 1, 2014 /PRNewswire/ -- Scioderm, Inc. today announced it has completed enrollment of its Phase 2b study (SD-003) to evaluate the safety and efficacy of SD-101, a novel topical therapy for the treatment of blisters and lesions in patients with Epidermolysis Bullosa (EB.) Key points: -- Epidermolysis Bullosa (EB) is a rare, genetic connective tissue disorder that typically manifests at birth or early childhood. -- All forms of EB share the common symptom...

2014-02-28 08:24:57

CAMBRIDGE, Mass., Feb. 28, 2014 /PRNewswire/ -- Zafgen, Inc., a leading biopharmaceutical company dedicated to addressing the unmet needs of severely obese patients, today announced it will join the National Organization for Rare Disorders (NORD) and others around the world in observing Rare Disease Day on February 28, 2014. On this day, millions of patients and their families will share their stories to focus a spotlight on rare diseases as a global public health concern. A rare...

2014-02-07 13:24:38

A genetic disorder that affects about 1 in every 2,500 births can cause a bewildering array of clinical problems, including brain tumors, impaired vision, learning disabilities, behavioral problems, heart defects and bone deformities. The symptoms and their severity vary among patients affected by this condition, known as neurofibromatosis type 1 (NF1). Now, researchers at Washington University School of Medicine in St. Louis have identified a patient’s gender as a clear and simple...

2014-01-22 08:28:52

DURHAM, N.C., Jan. 22, 2014 /PRNewswire/ -- Scioderm, Inc., today announced that the European Commission (EC) has granted orphan drug designation for SD-101 for the treatment of patients with Epidermolysis Bullosa (EB), a group of rare genetic skin diseases. This decision follows a positive opinion adopted by the Committee for Orphan Medicinal Products (COMP), recommending such designation. Epidermolysis Bullosa is a rare genetic connective tissue condition that, in all of its...

2014-01-15 23:01:02

Roger J. Packer, MD, and Yuan Zhu, PhD, named as the first two chairs to drive the transformation of care for children with Neurofibromatosis. Washington, DC (PRWEB) January 15, 2014 Children’s National Health System today installed Roger J. Packer, MD, and Yuan Zhu, PhD, as the inaugural Gilbert Family Professors in Neurofibromatosis. The professorships, funded through the generosity of Jennifer and Dan Gilbert, will allow Dr. Packer and Dr. Zhu to dedicate their time to advance the...

2014-01-15 08:27:42

Beloranib trial demonstrates improvements in body composition and cardiovascular risk factors in the largest placebo-controlled, double-blind, randomized multiple dose trial performed for obesity to date in patients with Prader-Willi syndrome CAMBRIDGE, Mass., Jan. 15, 2014 /PRNewswire/ -- Zafgen, Inc., a leading biopharmaceutical company dedicated to addressing the unmet needs of severely obese patients, today announced initial results from its Phase 2a study of beloranib, a...

2014-01-07 08:30:32

DURHAM, N.C., Jan. 7, 2014 /PRNewswire/ -- Scioderm, Inc., today announced that Dr. Robert Ryan, President and Chief Executive Officer of Scioderm, was invited to present at the 32nd Annual J.P. Morgan Healthcare Conference at 7:30 a.m. Pacific Standard Time (10:30 a.m. EST) on Thursday, January 16, 2014 in the Elizabethan C/D room of the Westin St. Francis hotel. Dr. Ryan will discuss the company's lead investigational therapy, SD-101, currently in late stage clinical development...

2014-01-06 08:26:03

DURHAM, N.C., Jan. 6, 2014 /PRNewswire/ -- Scioderm, Inc. today announced the initiation of a Phase 2B study designed to evaluate the efficacy and safety of SD-101, a novel topical therapy, for the treatment of non-healing wounds in patients with Epidermolysis Bullosa (EB), a rare, genetic connective tissue disorder that typically manifests at birth or early childhood. "People affected by EB suffer skin blisters, chronic wounds, almost constant pain and itching, and scarring," said...

2013-12-27 10:29:35

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear. In a study online December 26th in the ISSCR's journal Stem Cell Reports, published by Cell Press, researchers evaluated a patient with a genetic skin disorder known as epidermolysis bullosa (EB) nearly seven years after he had undergone a gene therapy procedure as part of a clinical trial. The study revealed that a small...

2013-12-23 23:22:39

Living With KP created by Jennifer Richards is a new book that teaches people how to get rid of their KP (Keratosis Pilaris) effectively. A detailed Living With KP review posted on the site HealthReviewCenter.com illustrates whether this book is worth buying. Seattle, Wa (PRWEB) December 23, 2013 Keratosis pilaris or follicular keratosis is a common skin condition, which is manifested by the appearance of rough, slightly red, and bumps on the skin. This disease appears on the back and...


Word of the Day
malpais
  • The ragged surface of a lava-flow.
'Malpais' translates from Spanish as 'bad land.'