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uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study With FIX-Padua Variant in 2018

October 26, 2017

~ AMT-060 with the FIX-Padua Modification (AMT-061) Demonstrates Substantial Increase in FIX Activity in Non-human Primates ~ ~ Plans to Initiate Pivotal Study with Enhanced AMT-061 in 2018 ~ ~ Achieves Alignment with FDA on Streamlined Clinical and Regulatory Strategy for AMT-061, Which Will be

Help Us Continue Our Success! Hear our Stories

A young man was in need of assistance with dental care. His dentist stated he would lose his teeth without repair immediately. BDASC stepped in to help cover some of the cost. Today, he smiles brightly because we were there to help.

"We just wanted to say thank you so much for a wonderful weekend and the opportunity to connect with other families. It was amazing! Our children made some new friends too and had a ball." "Keeping It Coastal, 2021 Educational Family Camp"

A member needed our help to advocate on his behalf when his access to his medication (factor) was in jeopardy. We were there to advocate on his behalf and today, he is doing well. "Hemophilia and Bleeding Disorders Advocacy Coalition"

When our teens come to the Teen Retreat, they learn independence and share accomplishments with each other about living with a bleeding disorder -one young lady learned how to self - infuse her medication with the encouragement of her friends. Now she is off to college and able to care for her own condition.

"We walk every year for hope. Hope that we will eventually see a cure. Hope that we will have better treatment options available to us. Hope that Daphne will grow up feeling confident and secure, even though she has something different about her. Hope that everyone with a bleeding disorder has access to care, education and advocacy. This is a hard road we walk every day, knowing that there is an organization out there to support us makes it a little easier".

I was around from time to time, and knew of a few things that were going on. With the VIP program, I know what is happening and when it is happening, and I can be involved like never before. I don’t think that I would be anywhere near in the know like I am now. I LOVE this program. Also, when I have a bleed or am laid up for a day or two, I can even sit down and watch a movie. THANK YOU VERY MUCH!!!

Bleeding Disorders Association of South Carolina welcomes you to register with our non-profit organization so we can keep you informed with information on events, advocacy initiatives, treatments therapies and research, fundraising opportunities, and educational and social networking opportunities. We invite those who are affected by bleeding disorders to register as well as individuals, families, friends and affiliates who are interested in supporting our organization and share our mission. You can download the registration form and fax, mail, or e-mail it to the Chapter, or you may register online. Welcome to Bleeding Disorders Association of South Carolina!

Bleeding Disorders Association of South Carolina is a 501(c)3 non-profit organization and a chapter of the National Bleeding Disorders Foundation and a member organization of Hemophilia Federation of America.

Our mission is to raise awareness for and advocate on behalf of persons with bleeding disorders and their families; provide education and supportive services; and promote ongoing research to improve the quality of life for those affected.

See our brochure (PDF) to learn more: DOWNLOAD

Brochure in Spanish: DOWNLOAD

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