An Opportunity for Caregivers

Please consider participating in this advisory panel to help bluebird bio to better understand ALD pre/during/post stem cell transplant.

bluebird bio plans to host a caregiver advisory board on November 7th in advance of the ALD Connect meeting in Waltham, MA. bluebird is looking for 6-8 caregivers of boys with ALD who have received allogeneic hematopoietic stem cell transplant (allo-HSCT) to gain insight and better understanding into the current allo-HSCT experience and unmet need, and perspectives on education gaps and current patient services for the community in the United States.
Please note that all interested individuals will go through a screening process and not all who are interested will qualify. For those who do qualify, an honorarium will be provided in compensation for their time as well as hotel accommodations for the night of November 6 and 7. For those participants who are not already anticipating attending the ALD Connect meeting, a travel stipend may be available.
For more information contact:
Liza Fiore (Snow Companies)


THIS is ALD #14 – Alex

We are finally caught up with the original stories and ready to keep going. Welcome to THIS is ALD #14 — Alex (and his family).

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My family knows ALD and it’s damned misfortunes too well. I lost a little brother, Miguel, in 2001 when he was only 10-years-old. We lived in Mexico where there is very little knowledge of ALD and unfortunately the doctors treated my brother with a series of “experiments”. He died as a result of the doctor’s negligence.


Last year our family received more terrible news. Doctors had once again had a difficult time diagnosing odd behavior and dark tanning (two symptoms connected to ALD) in my 8-year-old nephew, Angel, and my 7-year-old son, Alex. Both the boys tested positive for ALD.

Angel, deteriorated very quickly and does not speak, walk, move or eat on his own. He is also in diapers. Just a few weeks ago we got the news that his younger brother, Adam, also has the mutation. He is only 3-years-old. ALD is a cold and controlling disease.

After my son, Alex, was diagnosed, I shared information about what was happening with my son with Dr. Eichler at MassGeneral Hospital for Children. We were relieved when he and his team gave us the opportunity to be included in a gene therapy trial. We are currently in Boston for the treatment and feel very optimistic that the treatment will stop the progression of the disease.

—Griselda Jiménez


ALD is a cruel disease that can strike out of nowhere and move quickly through a family. As newborn screening becomes more and more common in the US, we are going to see many more “good ALD stories”, but we need to remember that there is a whole world out there that needs to be educated – about the disease itself, newborn screening and treatment options. Alex is blessed to have a family that managed to find one of the leading ALD doctors in the US and that Alex qualified for the gene therapy trial (for more about gene therapy and ALD – — CLICK HERE). Their family has been given the gift of hope.

Stay strong Alex! You are a warrior with a brilliant smile!!!! Sending love to you, your cousins and your entire family.

Love, Jess

Update: Alex got out of the hospital Monday and is doing great!