The nonprofit, Huntington’s Disease Society of America (HDSA) continues to regularly update information on research and advocacy. Consider signing up for the HDSA newsletter. It contains a wealth of information. http://hdsa.org.
Perhaps you are wondering about Right-to-Try legislation for Huntington’s disease patients who are end stage. HDSA provides a summary of the topic.
On its face, the idea behind Right-to-Try legislation is to allow families access to medications and treatments still in the clinical trial phase when an individual has no other treatment options left and is facing a terminal illness. Right-to-Try legislation focuses on cutting red tape in the Food and Drug Administration (FDA) to allow easier access to these experimental treatments. But, in reality, the FDA isn’t the issue. Access to investigational therapies is already permitted through the Food and Drug Administration’s expanded access program. FDA currently approves 99.7% of all expanded access requests submitted by physicians and companies for patients with immediately life-threatening illnesses who cannot participate in clinical trials. Removing FDA from the process of obtaining investigational drugs, as Right-to-Try proposes, increases the risk of patient harm and creates confusion. When access to an experimental drug or treatment is denied, it’s almost always because the request has been denied by the pharmaceutical company, not the FDA.”
At this time all Expanded Access and Right-To-Try requests to Roche will be denied. This was discussed in the recent HD Community-wide letter that was released by Roche in June. Specifically, they said “We understand that families may wish to seek access to investigational medicines as soon as possible. However, access to RG6042 can only be through clinical trial participation at this time. Because the benefits and risks of RG6042 are not fully understood, we are not able to grant pre-approval, compassionate use or “right-to-try” requests. The company Roche/Genentech
HDSA’s National Youth Alliance (NYA) motivates youth to get involved in their local Huntington’s Disease Society of America Chapter. Every child of a parent with HD has a 50/50 chance of inheriting the expanded gene that causes the disease. If the child has not inherited this expanded gene, he or she will never develop the disease and cannot pass it on to their children.
The Miller Family whose 3 children are involved in the NYA.
The NYA is made up of young women and men ages 9-29 from across the U.S. that are impacted by Huntington’s.
The NYA mission to not only support young people within the HD community, but also inspire the youth of HDSA to get involved in the battle against HD and be proactive in this fight. http://nya.hdsa.org/
Therese is the author of Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. 100% of the proceeds are being donated to HDSA. https://www.amazon.com/-/e/B06ZY85776
In the following video are the faces of Huntington’s disease (HD); a devil of a disease that attacks adults and children. Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It’s like having ALS, Parkinson’s and Alzheimers at the same time. HD is known as the quintessential family disease because every child of a parent with HD has a 50/50 chance of carrying the faulty gene. Today, there are approximately 30,000 symptomatic Americans and more than 200,000 at-risk of inheriting the disease. http://hdsa.org/what-is-hd/
Huntington’s Disease Society of America Annual Convention Continues:
Today, Friday, is the day we begin to explore Huntington’s Disease topics presented by experts/researchers/doctors in the HD world. The presenters travel from around the world to speak at this convention, so I feel very lucky to be attending.
At 9 a.m., the day will begin with the Opening Keynote Speaker.
By early morning, the attendees will select the Educational Workshops they wish to attend
Huntington’s Disease Society of America Annual Convention Continues:
Day 1. Leadership Day, for those in HDSA Chapters with Leadership roles, is today, June 7th. It will start off at 8 a.m. with a Continental Breakfast and at 9 a.m., Nancy Rhodes, Director of Field Development & Operations, and Bill Kline will be our first speakers. http://hdsa.org/about-hdsa/annual-convention/
After the meeting, at 6 p.m., there will be a Team Hope Walk for all to participate in. (You will receive a Team Hope Walk t-shirt.)
Then, the Convention Welcome Reception will begin at 7 p.m. and the Exhibit Hall will be open. (HDSA is graciously allowing me to sell my book again this year).
We Can Never Lose HOPE…………
HDSA Northern California Chapter Convention/Education Day
John and I are also on our way, in our RV; it’s about an eight hour drive from Northern California. We’ll be staying a Dockweiler State Beach; it has 3.7 miles of ocean frontage and 288 acres of beach and only four miles from the Marriott Convention Center where the Convention is being held.
This is my second Convention, and I was so impressed with HDSA staff, the support and hope that resonated throughout the three days last year. I truly believe this is my second family who understand the Huntington’s Disease journey because they are living it just as I did for 25 years with my three sisters-in-law, Lora, Marcia and Cindy Marin.
We Can NeverLose HOPE…………………
John, Dr. Vicki Wheelock, and myself at the 2017 HDSA National Convention in Schaumburg IL.
HOPE Walk held outside convention center at the 2017 HDSA National Convention in Schaumburg IL.
James and Mel Miller at the 2017 HDSA National Convention in Schaumburg IL.
California Resident Convention Scholarship- The intent of this scholarship is to help families and individuals who live in the state of California to attend the 33rd Annual HDSA Convention. The scholarship is open to any family member or caregiver who is affected by Huntington’s disease.
100% of the proceeds from Therese’s book sold in the U.S. will be donated to HDSA. http://www.hdsa.org Last December, Therese and John donated $9,015.00 to HDSA that was the profit from book sales since Therese published in April 2017.
I received this brochure in the mail from Huntington’s Disease Society of America (HDSA) the other day to register for this years National Convention in Los Angeles and I thought I’d share it. (See below to print a copy) I will be attending as a Board Member of the Northern California Chapter and I will also be selling my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. http://hdsa.org/about-hdsa/annual-convention/
Marjorie Guthrie’s Philosophy-Wife of Woody Guthrie who suffered from Huntington’s disease
“There must be a strong, well supported National organization to assure the widest possible distribution of all our educational materials to the HD families, doctors, neurologists…to the whole scientific community and health professionals everywhere. http://hdsa.org/about-hdsa/hdsa-history/
We must continue our search for HD families everywhere.
We must give active support to conferences dealing specifically with HD as well as related disciplines and disorders.
We must support and develop chapters in their local efforts and TOGETHER with National CCHD create the necessary financial resources to do the work that must be done.
We must begin a program, albeit limited at this time…to serve affected families wherever they may be….sometimes within the area of a chapter and when necessary beyond the capability of a chapter.
We must do everything possible to improve and expand our communications with our chapters…the public at large…..affected and non-affected families.
We must cooperate with existing health agencies and the federal, state and city government to improve the status of HEALTH and HEALTH PROGRAMS as a national priority.
All of these efforts must be done within the financial capability and responsibility of our membership.
Finally we must recognize that RESEARCH means many things. Any research in the areas of genetics and brain and central nervous system…however identified…may contribute ultimately towards a cure, control and early detection of HD.