Every hemo mom has the day she must send her child off on an overnight school trip, trusting that she has done everything she can to prepare her child for the responsibility of being on their own. My time came a few weeks ago when my son went to Orlando with his Sing Out […]
Dear Addy, I’m applying for life insurance, but I’m either ineligible or being told I have to pay three times the normal rate because I have hemophilia. Is this typical with a rare disorder, and is there anything I can do about it? Signed, Planning Ahead Dear Planning, Unfortunately, as you’ve found, your health status […]
Dimension Therapeutics announced it will discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of May 2017, and the observation that the data would not meet […]
Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.
uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting in Washington, D.C. on successful repeated hepatic gene delivery with their AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]
Kedrion Biopharma announced a return to normal supply levels of Koāte in the 1000 vial size and a series of large-scale improvements to their overall operations following a temporary period of limited availability of product. Click here to read the full contents of letter provided by Kedrion Biopharma regarding this matter.
Back in the “old days” before prophylaxis (prophy), hemophiliacs were treated on demand when they had a bleed. Max is just old enough to fall into that category so he didn’t switch to prophy until he was five years old. By then he had already had enough bleeds in his left ankle that the damage […]
When ABC premiered, “Speechless” last fall, I wasn’t sure if it would meet my expectations. Would it show special needs parenting and family life in a realistic, often sarcastic way, or would it have the feel of an afterschool special? I’m happy to say it’s readily become one of a handful of shows we watch […]
uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe […]
With summer just around the corner, we’ll soon be able to pull out those grills for our favorite outdoor meals. Whether you’ve got a taste for meat, fish, seafood, tofu or vegetables, grilling is one of the healthier cooking methods. Cooking food on a grill allows excess fat to drip away from the food, without […]
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