Krystal Biotech receives approval in Europe for gene therapy treatment
A gene therapy company has received a designation for one of its treatments from the European Medicines Agency.
Krystal Biotech, developing and commercializing novel treatments for dermatological diseases, announced that KB103, a treatment for a severe skin disorder, was granted an Orphan Medicinal Product Designation by the agency, according to a news release.
In November 2017, the FDA granted the same de signation for the treatment, which is meant for dystrophic epidermolysis bullosa, an incurable… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 19, 2018 Category: Biotechnology Authors: Stacey Federoff Source Type: news
Abeona Therapeutics Appoints Carsten Thiel, Ph.D., as Chief Executive Officer
Company strengthens management team with biopharmaceutical veteran bringing extensive commercial experience in life-threatening rare diseases
Tim Miller, Ph.D. continues as president and assumes chief scientific officer role
NEW YORK and CLEVELAND, A... Biopharmaceuticals, Personnel Abeona Therapeutics, gene therapy, epidermolysis bullosa, Sanfilippo syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 2, 2018 Category: Pharmaceuticals Source Type: news
Queensland man with a severe skin condition defies doctors
Dean Clifford, from Queensland, has become the oldest living survivor in the world of the most serious form of Epidermolysis bullosa, which strikes one in 50,000 people. (Source: the Mail online | Health)
Source: the Mail online | Health - March 15, 2018 Category: Consumer Health News Source Type: news
Man, 38, with a severe skin condition defies doctors
Dean Clifford, from Queensland, has become the oldest living survivor in the world of the most serious form of Epidermolysis bullosa, which strikes one in 50,000 people. (Source: the Mail online | Health)
Source: the Mail online | Health - March 15, 2018 Category: Consumer Health News Source Type: news
Raquel Hultman has skin syndrome that tears at the slightest touch
Raquel Hultman, eight, was diagnosed with epidermolysis bullosa (EB) at birth, a rare genetic tissue disorder which has no cure, but she loves to ski in a thick padded jacket that protects her (Source: the Mail online | Health)
Source: the Mail online | Health - March 9, 2018 Category: Consumer Health News Source Type: news
Little girl has skin syndrome that tears at the slightest touch
Raquel Hultman, eight, was diagnosed with epidermolysis bullosa (EB) at birth, a rare genetic tissue disorder which has no cure, but she loves to ski in a thick padded jacket that protects her (Source: the Mail online | Health)
Source: the Mail online | Health - March 9, 2018 Category: Consumer Health News Source Type: news
'Butterfly disease' sufferer refuses life-saving treatment
WARNING: GRAPHIC CONTENT Jonathan Gionfriddo, 17, from Stafford Springs, Massachusetts, endures agonising pain every day due to epidermolysis bullosa. (Source: the Mail online | Health)
Source: the Mail online | Health - January 29, 2018 Category: Consumer Health News Source Type: news
Grow your own: the race to create body parts in the lab
From replacement skin to entire new organs, regenerative medicine is finally leaving its early scandals – and the controversial ‘earmouse’ – behind. Could it one day provide a cure for birth defects, blindness and diabetes?Two years ago, Hassan ’s father was faced with questions that he had no good answers for. “Why do I have this disease?” his seven-year-old son asked him. “Why do I have to live this life?”Hassan was born with a rare genetic skin condition, called epidermolysis bullosa, that causes fragile, blistering skin. His first blister appeared when he was a week old, but soon after his family fled...
Source: Guardian Unlimited Science - December 5, 2017 Category: Science Authors: Hannah Devlin Tags: Medical research Health Doctors Society Stem cells Diabetes Biology Science Source Type: news
Tia has epidermolysis bullosa - known as 'butterfly skin'
Four-year-old Tia is hoping for pioneering treatment for her fragile skin condition. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - November 16, 2017 Category: Consumer Health News Source Type: news
Stem Cell Gene Therapy Replaces Boy's Entire Epidermis Stem Cell Gene Therapy Replaces Boy's Entire Epidermis
Ex vivo gene therapy of epidermal cells from a boy with junctional epidermolysis bullosa enabled grafts to completely replace his epidermis.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - November 9, 2017 Category: Consumer Health News Tags: Dermatology News Source Type: news
'Butterfly' boy saved after doctors create him a new skin
WARNING, GRAPHIC CONTENT: The unnamed boy, who suffered from junctional epidermolysis bullosa, was treated by doctors at Bochum University Hospital in Germany. (Source: the Mail online | Health)
Source: the Mail online | Health - November 8, 2017 Category: Consumer Health News Source Type: news
Genetically Altered Skin Saves A Boy Dying Of A Rare Disease
An inherited disease called epidermolysis bullosa destroyed the 7-year-old's skin. Scientists grew new skin in the lab without the genetic flaw, and replaced 80 percent of his skin. He is thriving.(Image credit: CMR Unimore/Nature ) (Source: NPR Health and Science)
Source: NPR Health and Science - November 8, 2017 Category: Consumer Health News Authors: Richard Harris Source Type: news
Gene Therapy Creates Replacement Skin to Save a Dying Boy
Doctors grew sheets of healthy skin that were transplanted onto a boy with a genetic disease that caused blistering and tearing all over his body. (Source: NYT Health)
Source: NYT Health - November 8, 2017 Category: Consumer Health News Authors: DENISE GRADY Tags: Skin Epidermolysis Bullosa Genetic Engineering Nature (Journal) Source Type: news
Gene Therapy Creates New Skin to Save a Dying Child
Doctors grew sheets of healthy skin that were transplanted onto a boy with a genetic disease that caused blistering and tearing all over his body. (Source: NYT Health)
Source: NYT Health - November 8, 2017 Category: Consumer Health News Authors: DENISE GRADY Tags: Skin Epidermolysis Bullosa Genetic Engineering Nature (Journal) Source Type: news
Genetically modified skin grown from stem cells saved a 7-year-old boy ’ s life
Scientists reported Wednesday that they genetically modified stem cells to grow skin that they successfully grafted over nearly all of a child's body — a remarkable achievement that could revolutionize treatment of burn victims and people with skin diseases. The research, published in the journal Nature, involved a 7-year-old boy who suffers from a genetic disease known as junctional epidermolysis […]Related:She signed up to be a surrogate mother — and unwittingly gave away her own childWhite House opioid commission calls for wide-ranging changes to anti-drug policiesScreams, torture and so much blood: The...
Source: Washington Post: To Your Health - November 8, 2017 Category: Consumer Health News Source Type: news
