The Wiskott-Aldrich Foundation

Happy Father's Day to all of the amazing dads living with Wiskott-Aldrich Syndrome and those who care for sons with WAS! Your strength, resilience, and unwavering love inspire us every day. Today, we celebrate you and the special role you play in your families' lives.

On this day, join the Wiskott-Aldrich Foundation to remember and honor those who have sacrificed.

Happy Mother's Day 💙 to all of the extraordinary mothers in our community who devote themselves tirelessly to caring for their children with Wiskott-Aldrich Syndrome. You are the pillars of support for our families, showing strength, resilience and unconditional love through all of the challenges of WAS.

In honor of those who selflessly sacrificed for their loved ones and tragically lost them, we hold close the memory of your boundless love, the courage and unwavering commitment you showed at the darkest of times. You continue to inspire us, and your love echoes through the hearts of our community.

Thank you for all that you do and the sacrifices you make. Happy Mother's Day! 💙💙💙💙

REMINDER!
REGISTRATION REQUIRED!
Please click here to register: https://forms.gle/Pg5AyfbFDrqfUxKE7
Dr. Michael Albert et al have published a groundbreaking study of 577 patients showing a correlation between the WAS mutation and the severity of the disease. One of the findings show that patients with XLT/Mild WAS tend to develop the same complications as those with Severe WAS, just a bit later in life and that they are at risk for premature mortality. Here is a link to the paper. https://doi.org/10.1182/blood.2023021411

Please join us for a talk by Dr. Albert sharing the findings of his paper:
Wiskott-Aldrich Syndrome: "Relationship between mutation and the severity of disease. XLT is NOT a mild disease!"

When: May 10, 8:00pm (Germany), 9:00 PM (Israel) 7:00pm (United Kingdom), 2:00pm (Eastern USA), 1:00pm (Central USA), 12:00pm (Mountain USA), 11am (Pacific USA), 4am (Australia)
Where: Virtual Meeting on Zoom
Please click here to register: https://forms.gle/Pg5AyfbFDrqfUxKE7
Who can attend: Anyone over the age of 18 years who has been impacted by WAS/XLT. If you have questions about whether your child's mutation is mild or severe, what course to expect based on the mutation, what complications to expect and when do they occur, whether to transplant or not, how do WAS patients fare after transplant/gene therapy. This is a chance to bring all these questions for the WAS Expert.

What is the talk about?
How to correlate the WAS mutation to the expected severity of the disease
Complications of Severe WAS/XLT: Bleeding, infection, autoimmunity, malignancy, at what ages do they happen in Severe WAS, in XLT and the severity.
Prognosis for patients with XLT/Mld WAS with and without transplant
Outcome of HCT and GT for mild and severe WAS
New proposed management guidelines
Please join us for this important talk. This is an excellent opportunity to bring the many questions we all have to ask the expert. Please let us know if you have any questions.
Looking forward to seeing everyone at the meeting. You'll receive the link to the meeting two days prior.

REGISTRATION REQUIRED!
Please click here to register: https://forms.gle/Pg5AyfbFDrqfUxKE7

Dr. Michael Albert et al have published a groundbreaking study of 577 patients showing a correlation between the WAS mutation and the severity of the disease. One of the findings show that patients with XLT/Mild WAS tend to develop the same complications as those with Severe WAS, just a bit later in life and that they are at risk for premature mortality. Here is a link to the paper. https://doi.org/10.1182/blood.2023021411

Please join us for a talk by Dr. Albert sharing the findings of his paper:

Wiskott-Aldrich Syndrome: "Relationship between mutation and the severity of disease. XLT is NOT a mild disease!"

When: May 10, 8:00pm (Germany), 9:00 PM (Israel) 7:00pm (United Kingdom), 2:00pm (Eastern USA), 1:00pm (Central USA), 12:00pm (Mountain USA), 11am (Pacific USA), 4am (Australia)

Where: Virtual Meeting on Zoom

Please click here to register: https://forms.gle/Pg5AyfbFDrqfUxKE7

Who can attend: Anyone over the age of 18 years who has been impacted by WAS/XLT. If you have questions about whether your child's mutation is mild or severe, what course to expect based on the mutation, what complications to expect and when do they occur, whether to transplant or not, how do WAS patients fare after transplant/gene therapy. This is a chance to bring all these questions for the WAS Expert.

What is the talk about?
How to correlate the WAS mutation to the expected severity of the disease
Complications of Severe WAS/XLT: Bleeding, infection, autoimmunity, malignancy, at what ages do they happen in Severe WAS, in XLT and the severity.
Prognosis for patients with XLT/Mld WAS with and without transplant
Outcome of HCT and GT for mild and severe WAS
New proposed management guidelines

Please join us for this important talk. This is an excellent opportunity to bring the many questions we all have to ask the expert. Please let us know if you have any questions.

Looking forward to seeing everyone at the meeting. You'll receive the link to the meeting two days prior.

World PI (primary immunodeficiency) Week is a global campaign to raise awareness and improve diagnosis and treatment of primary immunodeficiencies. Wiskott Aldrich Syndrome (WAS) is a rare primary immunodeficiency that affects mostly males (rarely females) around the world. Symptoms can range from mild to severe. Low platelets, eczema, upper respiratory infections, ear infections, and immune system deficiencies are some of the symptoms. Bone marrow transplants and experimental gene therapy are the definitive treatments. however, those are done with significant side effects and risk. It is our hope to raise awareness of WAS so early diagnosis can occur and less toxic treatments can be developed.

World PI (primary immunodeficiency) Week is a global campaign to raise awareness and improve diagnosis and treatment of primary immunodeficiencies. Wiskott Aldrich Syndrome (WAS) is a rare primary immunodeficiency that affects mostly males (rarely females) around the world. Symptoms can range from mild to severe. Low platelets, eczema, upper respiratory infections, ear infections, and immune system deficiencies are some of the symptoms. Bone marrow transplants are the only cure, however, those are done with side effects and risk. It is our hope to raise awareness of WAS so early diagnosis can occur and less toxic treatments can be developed.

🌟 Big News! 🌟

The Wiskott-Aldrich Foundation is thrilled to extend our deepest gratitude to the Telethon Foundation for their unwavering dedication to advancing gene therapy for Wiskott-Aldrich Syndrome patients. 🙌 Together, we're breaking barriers and paving the way for life-changing treatments. 💪 Thank you, Telethon Foundation, for joining us on this incredible journey!

🧬❤️

📢 Grandi notizie dalla Fondazione Telethon. Un altro passo verso il futuro!
🔬Fondazione Telethon annuncia l’impegno a rendere disponibile ai pazienti la terapia genica per la sindrome di Wiskott-Aldrich.
🔬Si tratta della seconda terapia genica abbandonata dall’industria di cui la Fondazione Telethon si fa carico affinché arrivi ai pazienti che ne hanno bisogno.
👉https://www.telethon.it/storie-e-news/news/dalla-fondazione/fondazione-telethon-annuncia-limpegno-a-rendere-disponibile-ai-pazienti-la-terapia-genica-per-la-sindrome-di-wiskott-aldrich/?utm_source=facebook&utm_medium=social_organic

As we approach the end of the year, let's make a big impact for those affected by Wiskott-Aldrich Syndrome. Please extend your support to those affected by this rare immunodeficiency disorder by making a donation to the Wiskott-Aldrich Foundation. Every contribution, no matter the size, plays a crucial role in improving the lives of those living with WAS.

Let's end the year on a compassionate note by spreading love and hope to those that need it.

1. Donate by going to wiskott.org and click the DONATE button.
2. Or scan the QR code on the image

Together we can make a lasting impact!

The Wiskott Aldrich Foundation is a 501(c)(3) public charity. All donations are tax deductible.

On February 3, 2023, the Wiskott-Aldrich Foundation convened the Externally Led-Patient Focused Drug Development (EL-PFDD) meeting to educate the FDA and other stakeholders on how X-Linked Thrombocytopenia and Wiskott-Aldrich Syndrome (WAS) impacts our lives, and give our thoughts on current and future therapies. More than 300 individuals participated in the live webcast event, and patients and family members testimony has been captured in the 𝐕𝐨𝐢𝐜𝐞 𝐨𝐟 𝐭𝐡𝐞 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐑𝐞𝐩𝐨𝐫𝐭. This landmark report documents the severe disease burden and unmet medical need in patients' own voices.

The Wiskott-Aldrich Foundation wants the FDA and all the stakeholders to listen to our patients' voice, their words, and their experiences, and recommends that these findings be considered when developing potential XLT/WAS therapeutics. The 𝐕𝐨𝐢𝐜𝐞 𝐨𝐟 𝐭𝐡𝐞 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐑𝐞𝐩𝐨𝐫𝐭 has been submitted to the FDA for inclusion in the framework used to evaluate future therapies for XLT and WAS. The report is here: https://www.wiskott.org/pfdd

The report, video of the live webcast, are posted below. Our community owes a deep debt of gratitude to the patients and family members who courageously shared their stories, took the time to attend the meeting and to answer questions about how XLT/WAS has impacted their lives and what improvements they would like to see. https://www.wiskott.org/pfdd

This year, several Wiskott Warriors died. Our hearts go out to the families who have lost their sons, husbands, fathers, nephews, and friend. What causes this to happen? First of all, there are no safe treatments available. There are serious risks associated with bone marrow transplants, including death. In addition, Wiskott-Aldrich Syndrome can cause life-threatening bleeding, infections, and autoimmunity. Once these symptoms manifest, medical treatments may not be adequate.

Families are often left with financial hardships after losing a loved one, such as hospital bills, unemployment, and funeral costs. In addition, they suffer from deep grief.

It is the mission of the Wiskott-Aldrich Foundation to provide support and community to these families. The Foundation is also on a mission to fund medical research that can lead to cures. The Foundation needs your help to remain strong and viable. During this , please consider making a donation to the foundation. Every dollar counts.

Please submit a donation on our secure website: https://www.wiskott.org/About-WAS/donate

We need to help these Wiskott Warriors fight their battles!

The Wiskott Aldrich Foundation is a 501(c)(3) public charity. All donations are tax deductible.
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As a 100% volunteer organization, the Wiskott-Aldrich Foundation is funded and operated by donors. Due to the rare nature of Wiskott-Aldrich Syndrome, the foundation has had difficulty obtaining funding and support. Our most important source of funding is the generosity of donors like you.

This 2023 is another chance to help us save as many lives as possible. This money is used to provide hardship grants to families in need around the world, as well as to support research into cures throughout the global medical community.

To extend the lives of the boys born with Wiskott-Aldrich Syndrome, we need your help. Please give what you can. Every little bit counts!

Easily donate by scanning the secure QR Code on the image or click on our secure donation link here:
https://www.wiskott.org/About-WAS/donate

The Wiskott Aldrich Foundation is a 501(c)(3) public charity. All donations are tax deductible.
❤️

On behalf of The Wiskott-Aldrich Foundation, we wish you a Happy Thanksgiving 2023.

Please join us for the IDF National Conference, June 22-23, 2023.

There are many exciting and informative talks ranging from basics of immunology, to in depth medical and scientific talks, life management topics and disease specific talks. These are presented by expert immunologists and life management experts.

Included in the agenda is an exciting talk on Gene Editing for WAS by Dr. Matthew Porteus from 1 PM - 2 PM EDT on Thursday, June 22.

Please take the time to visit the exhibits including one from the Wiskott-Aldrich Foundation.

The meetings are virtual and there is no cost to attend.

To Register, please go to: https://web.cvent.com/event/329f0ba9-f21d-4604-a2d8-22c9360bb953/websitePage:cdef3525-0b3c-49fd-b13b-c887dd6940a7

For the agenda, please go tohttps://primaryimmune.org/sites/default/files/2023AgendaUpdated060223.pdf

It is time to discuss Wiskott-Aldrich Syndrome (WAS). It is a rare primary immunodeficiency affecting males. Symptoms include low platelets, infections, immunodeficiency, lymphoma, eczema, ear infections, sepsis, and painful cellulitis.

The importance of early diagnosis of WAS cannot be overstated, but it can be challenging especially if there is no family history of the disease or if there is a lack of information, which is why we need your assistance to spread awareness of WAS during World Primary Immunodeficiency Week (April 22-29). This is

Join us today and help us make a difference and advance research for all those affected by WAS. We, as a community, must improve diagnosis, treatment, and quality of life for families affected by WAS.

With your gift today, you will enable us to advance research and assist families facing medical hardships. Please consider making a donation through our secure donor website:
https://interland3.donorperfect.net/weblink/weblink.aspx?name=E340959&id=5

Or, if you prefer, checks can be written to:
Wiskott-Aldrich Foundation
PO Box 156
Austell, GA 30168

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Attention parents, caregivers, and WAS/XLT patients!

You have until March 8th to submit your comments for our EL-PFDD (Externally-Led Patient Focused Drug Development Meeting) Voice of the Patient Report.

Click on the Submit Comments link at https://www.wiskott.org/pfdd and answer the questions.

Sharing your experience and concern is important. Your voice is important for the Voice of the Patient Report, which will be available to the FDA, scientists, researchers, and industry.

WiskottAldrichSyndrome

Wiskott-Aldrich Syndrome (WAS) is a rare genetic immunodeficiency that affects the immune system. Additionally, it makes it difficult for a child's bone marrow to produce platelets, resulting in bleeding. Most of the cases occur in males.

Wiskott-Aldrich Syndrome is characterized by a deficiency of the immune system, eczema (an inflamed skin condition), and a reduced capacity to clot blood (low platelets).

We need your help to spread awareness of WAS and find better treatments.

The Wiskott-Aldrich Foundation would like to invite parents, caregivers, and WAS/XLT patients (over age 18) to participate in an informal follow-up to the EL-PFDD meeting we had last week, Feb 3. The purpose of this follow-up meeting is to continue the discussion, answer questions, and any concerns you might have after attending the EL-PFDD meeting.

Please register to attend the meeting.
https://forms.gle/6Ps4GYVndazqs4Hv6

When: February 16, 2023 at 4 pm - 5 pm EST.
Where: Virtually on Zoom (we will email the link to you)
Who: This meeting is for families and patients with Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia. Families of patients who have been cured are welcome to attend. You have to be over the age of 18 to attend.

RSVP by February 15, 2023.
https://forms.gle/6Ps4GYVndazqs4Hv6

Looking for true love this Valentine's Day? The CGDAssociation of America, in partnership with the The Wiskott-Aldrich Foundation and SCID, Angels for Life Foundation, would like to invite you to join a Self-Love Webinar.

When: Feb 9, 2023, 8:00PM Eastern Time
Register: http://bit.ly/cgd-love

Join Felicia Morton and Nicole Dauz, self-care coach and author, as they explore ways to show ourselves more love and self-care - no matter where you are on your rare journey. Join the CGD Association of America, Wiskott-Aldrich Foundation, and SCID Angels For Life Foundation to take part in a free, fun, and inspiring webinar, which is designed to encourage you to prioritize your most primary relationship: the one you have with yourself.

Nicole Dauz, self-care coach, certified meditation teacher, author, and mother to a daughter with disabilities, will join us to lead this inspiring workshop.

Everyone in the rare, primary immune community is welcome!

WAF EL-PFDD MEETING TODAY! FEBRUARY 3 @ 10AM-3PM EST.
LIVE LINK: https://www.wiskott.org/pfdd
POLLING QUESTIONS (USE CELL PHONE RECOMMENDED) - We need a lot of people to participate in the POLLING QUESTIONS: https://pollev.com/waspfdd
PHONE-IN LIVE: CALL 1-703-844-3231 when prompted

This Friday, Feb 3 at 10:00am EST is the WAF Externally-Led Patient Focused Drug Development Meeting for Wiskott-Aldrich Syndrome/XLT.
Register and Submit Comments Here: https://www.wiskott.org/pfdd

We have 20 families sharing their stories, their journeys, transplant survivors, GVHD survivors, and gene therapy success stories. Come join us to hear their stories.

We also need your input, we need to hear your voices! We'll have call-in and polling questions throughout the meeting. We need people to register and attend. Look at the agenda and see what hour you can attend and participate.

This is a once-in-a-life-time opportunity. It's open to everyone. Invite your family, friends, grandparents, teachers, therapists, and caregivers.

Register and Submit Comments Here: https://www.wiskott.org/pfdd

The Wiskott-Aldrich Foundation (WAF) requests your participation in our upcoming Externally-Led Patient Focused Drug Development Meeting. Our goal is to hear from patients and care-givers what it's like to live with Wiskott-Aldrich Syndrome/XLT (WAS/XLT) so the FDA, pharmaceutical companies, doctors, scientists, researchers, and other stakeholders can understand the patient experience. The information can help the FDA make informed decisions on approvals of potential medicines and therapies for WAS/XLT, and for companies and scientists to design clinical trials that are meaningful to WAS/XLT patients.

Register Here: https://www.wiskott.org/pfdd

When: February 3, 2023 @ 10:00AM-3:00PM EST
Where: Live online at https://www.wiskott.org/pfdd
Who Can Register: Anyone who lives with or is interested in WAS/XLT, patients, care-givers, families, and friends - are welcomed to attend.

Register Here: https://www.wiskott.org/pfdd

We'll hear stories from families and patients, and there will be open discussions where we can all join in on what it is like for us, as patients and caregivers to live with WAS/XLT and what we think of the current treatments. We can call in with our experience on the day of the meeting. We can also write in our comments in the registration form.

The more the voices that join in to share their stories, the more impact we will have. Please take the time to join us.

Please join us for the informational WAF EL-PFDD (Externally-Led Patient Focused Drug Development) Community Webinar on Thursday, January 12, 2023 from 4:00-5:00 PM EST. We really need to hear from Patients and Parents! We need at least 100 people to sign up. Please share this with your friends and family.

Register Here: https://forms.gle/d8Zxgm1z6g3EkUCc7

WAS, as we know, is an extremely challenging disease with only one cure, BMT, which is dangerous and toxic. After 11 years of success, gene therapy trials have been halted due to a lack of funding. Patients with XLT have even fewer options. As patients, as parents, as siblings, and as grandparents, we must speak up and share our experiences in order to get better cures sooner and ensure gene therapy trials are restored.

To address this we are hosting a one-hour community webinar, where you will learn about the drug development and research process as well as the benefits of the upcoming WAF's Patient-Focused Drug Development (PFDD) meeting on February 3, 2023 (mark your calendars for this important meeting as well).

The January 12th community webinar is designed to draw your attention to the important virtual meeting culminating for the Wiskott-Aldrich Syndrome community taking place on Friday, February 3, 2023 from 10am-3pm (mark your calendars now).
If you would like to learn more about the PFDD, here is a two-page simple summary: https://www.fda.gov/media/160223/download

There is no cost to attend but you need to pre-register for the community webinar ASAP. You must be 18 years or older to register. This webinar is for families, friends, and patients with Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia. Families of patients who have been cured are also encouraged to attend.

We need you to Register Here: https://forms.gle/d8Zxgm1z6g3EkUCc7

The Wiskott-Aldrich Foundation wishes you a happy 2023! Wishing you a healthy, happy, and fulfilling year filled with love, joy, and peace.

Wiskott-Aldrich Foundation's 20th Anniversary Gala was a big success in 2022. However, there is still a need for $20,000. As previously announced, we are pursuing efforts to bring Gene Therapy and other lifesaving therapies, such as Gene Editing, to the U.S. While the Anniversary Gala raised significant funds for this initiative, your support is still needed.

Please support the Wiskott-Aldrich Foundation with a gift today to help us finish strong.

This year the WAF is accepting donations through our secure donor website:
https://interland3.donorperfect.net/weblink/weblink.aspx?name=E340959&id=5

Alternatively, you can simply scan the QR code in the image and make your donation.
Or, if you prefer, checks can be written to:
Wiskott-Aldrich Foundation
PO Box 156
Austell, GA 30168

For 20 years, the Wiskott-Aldrich Foundation has supported families with medical grants. In the midst of a very difficult time in their child's life, these families experienced some financial relief. The donations you make to the WAF make a significant difference in the lives of WAS families.

Please support the Wiskott-Aldrich Foundation with a gift today to help us finish strong.

This year the WAF is accepting donations through our secure donor website:
https://interland3.donorperfect.net/weblink/weblink.aspx?name=E340959&id=5

Alternatively, you can simply scan the QR code in the image and make your donation.
Or, if you prefer, checks can be written to:
Wiskott-Aldrich Foundation
PO Box 156
Austell, GA 30168

Your year-end gift to the Wiskott-Aldrich Foundation (WAF) will help tackle the development of new treatments and lifesaving therapies for Wiskott-Aldrich Syndrome (WAS).

The WAF is currently conducting a Patient Focused Drug Development (PFDD) program to ensure the voices of WAS patients are heard and meaningfully incorporated into drug development and treatments. There is a significant amount of funding needed for this initiative.

Your gift will make a lasting impact for patients living with WAS, give hope to their situation.

We hope you will consider making a gift at one of the following levels at our website https://www.wiskott.org/:

$5,000
PFDD SPONSORSHIP VOICE OF THE PATIENT (VoP) REPORT
The VoP Report is a landmark document that summarizes the proceedings of the PFDD. It documents severe disease burden and unmet medical needs in the patients' own voice. The FDA and stakeholders use the document when considering the application and approval of newer therapies.

$2,000
1 FAMILY OUT-OF-POCKET EXPENSES DURING TREATMENT
Families face incredible financial strain with one parent having to give up their job on top of the already mounting bills.

$1,000
PROVIDE LIFE-SAVING MEDS OR CRITICALLY NEEDED SPECIALIZED HYPOALLERGENIC INFANT FORMULA
WAS is a life threatening disease. Unfortunately, some of the medications and critically important hypoallergenic infant formulas are not covered by insurance.

$500
FUNDS 1 GLOBAL EDUCATIONAL SESSION
WAS being a rare disease, there is a lack of up-to-date information. We host no-cost monthly educational sessions. Your support of $500 helps sponsor one educational session for our families.

This year the WAF is only accepting donations through our donor perfect website:
https://interland3.donorperfect.net/weblink/weblink.aspx?name=E340959&id=5
Alternatively, you can simply scan the QR code in the image and make your donation.
Or, if you prefer, checks can be written to:
Wiskott-Aldrich Foundation
PO Box 156
Austell, GA 30168