The Wiskott-Aldrich Foundation

10/09/2026

💙 Thank you, RARE Revolution Magazine! 💙

The Wiskott-Aldrich Foundation is grateful to Rare Revolution Magazine for shining a light on our Foundation and the Wiskott-Aldrich Syndrome community through such a thoughtful and beautifully written article.

We truly appreciate the care taken to share our story, our mission, and the experiences of the families who inspire our work every day.

Thank you for bringing more awareness to the WAS community and the rare disease community. We are honored to have our story shared with your readers.

💙 Read the full article: Heart, Soul & Glue 💙
https://bit.ly/RARE-X-Linked-HeartSoulAndGlue

💙 Read the full issue: Rare Revolution Magazine 💙 https://lnkd.in/euZgjTGU

Rare Revolution - Autumn 2026_Issue 037

10/08/2026

💙 Congratulations to our Co-Founder and Executive Director, Sumathi Iyengar! 💙

The Wiskott-Aldrich Foundation is proud to congratulate Sumathi on receiving the 2026 Abby Meyers Khushi Bridging RARE Award from Indousrare and Global Organization for Rare Disease (GORD).

For more than two decades, Sumathi has been a passionate advocate for the Wiskott-Aldrich Syndrome community, helping build bridges among families, clinicians, researchers, industry, and regulators from all over the world.

This well-deserved recognition honors not only her passionate dedication and leadership, but the impact of patient advocacy in advancing hope and meaningful progress for the Wiskott-Aldrich Syndrome community.

We are grateful for everything Sumathi has done and we wish her a big congratulations on receiving this award! 💙

IndoUSrare & GORD announce 2026 Abbey Meyers Khushi Bridging RARE Award honorees! Join us Nov. 1, Manassas, VA for an inspiring Gala

10/06/2026

We're so grateful to CNBC Cures for highlighting this important milestone for the Wiskott-Aldrich Syndrome community. Thank you for shining a light on WASKYRA™, UCSF Benioff Children's Hospitals, and the progress being made to expand access to this important treatment for people living with Wiskott-Aldrich Syndrome.

We're proud to be part of this growing community and excited about what's ahead for families affected by WAS.

Link to the CNBC newsletter: https://link.cnbc.com/public/47760577

Sign up for the CNBC Newsletter: https://www.cnbc.com/cnbc-cures-newsletter/?__source=newsletter%7Ccnbccures%7C20260911

10/06/2026

Great news for the The Wiskott-Aldrich Foundation and the families it serves!

CNBC Cures recently highlighted an important milestone: the opening of the first qualified treatment center for Wiskott-Aldrich syndrome, an exciting step forward in expanding access to specialized care for those living with this rare genetic disorder.

This progress makes us especially proud to celebrate Mike Skrynecki, CFP® RICP®, of Skrynecki Financial Planning Group of Raymond James in Atlanta, GA, our 2026 Invest in Others Lifetime Achievement Award recipient, whose longstanding dedication to the Wiskott-Aldrich Foundation earned the organization a $100,000 grant from Invest in Others this year.

Mike’s commitment is a powerful example of what can happen when financial advisors use their time, leadership, and resources to champion causes that are deeply personal to them and help create meaningful change for others.

Read more from CNBC Cares about the First Qualified Treatment Center Launching for Wiskott Aldrich Syndrome: https://lnkd.in/gn5JmNmu

You can also read Mike’s inspiring story and learn more about his commitment to changing the future for families living with Wiskott-Aldrich syndrome here:
https://lnkd.in/gY_YWf5a

We can’t wait to see how the Wiskott-Aldrich Foundation builds on this momentum and continues changing lives.

10/01/2026

Living with a rare immune disorder affects far more than physical health. Whether you are a patient, parent, caregiver, or family member, the journey can bring uncertainty, stress, fear, isolation, and emotional challenges at many different stages.

Please join the Wiskott-Aldrich Foundation, the SCID Foundation and the Hyper IgM Foundation for a special patient and family education webinar:

Mental Health & Rare Disease: Tools for Coping, Healing, and Connection

Saturday, October 24, 2026
11:00 AM–12:00 PM EDT

Register here:
https://forms.gle/sFGA2znDvBLMuWE38

We are delighted to welcome Ms. Julie Wells, Director of Strategic Relationships at Give an Hour, a national mental health nonprofit that works alongside rare disease communities.

Julie brings more than 32 years of experience in the nonprofit sector and has worked extensively with individuals and communities facing significant life challenges. She also has a deeply personal connection to the rare disease community through her own rare genetic heart condition, which affects several members of her family. In addition, Julie is a caregiver and a peer-support facilitator for caregivers and rare disease organization leaders.

During this interactive session, Julie will explore the connection between emotional and physical health and share practical, realistic tools that patients and families can use to care for themselves and support one another. We will talk about coping with the emotional impact of rare disease, building resilience and connection, and finding ways to improve overall well-being and quality of life.

The session will be moderated by Prof. Fabio Candotti, Head of Immunity at Lausanne University Hospital and the University of Lausanne, Switzerland.

This webinar is intended for adult patients, parents, caregivers, and family members (18 years and older) from the WAS, SCID, and Hyper IgM communities. Whether you are newly diagnosed, living with your condition without definitive treatment, preparing for HSCT or gene therapy, or many years beyond treatment, we hope this conversation will offer useful tools and support for wherever you are in your journey.

There will be ample time for questions and discussion, and we encourage you to submit your questions for Julie when you register.

There is no cost to attend.
RSVP by October 23, 2026.

Register here:
https://forms.gle/sFGA2znDvBLMuWE38

Registered participants will receive the Zoom information by email a few days before the webinar.

We hope you will join us for this important conversation. Caring for our mental health is an important part of caring for ourselves and our families throughout the rare disease journey.

09/28/2026

💙 A truly historic day for the Wiskott-Aldrich syndrome community!

UCSF Benioff Children’s Hospitals has been designated as the first U.S. Qualified Treatment Center for WASKYRA®, making this gene therapy commercially available to eligible patients in the United States.

It is difficult to put into words what this moment means.

For so many years, our community watched this therapy develop—from the earliest clinical trials, through changes and uncertainty about its future, to FDA approval in December 2025. Today, we have reached another extraordinary milestone: eligible patients in the U.S. can now access this treatment.

This achievement took an incredible village. Our deepest gratitude to Fondazione Telethon and SR-TIGET, Orphan Therapeutics Accelerator and Orphan Therapies, UCSF Benioff Children’s Hospitals, the researchers and clinicians who devoted years to developing and advancing this therapy, the FDA, members of the board of the Wiskott-Aldrihc Foundation, and everyone who worked to bring WASKYRA across the finish line and make it accessible to patients.

The The Wiskott-Aldrich Foundation is honored to have been an integral part of the team that made this happen. It's been a privilege working with all involved for the last 16 years.

Above all, we honor the patients and families who participated in the clinical trials. Their courage and willingness to step into the unknown helped make this day possible for every family that follows.

WASKYRA is also remarkable as the first advanced therapy to be commercialized entirely through a nonprofit collaboration—a powerful example of what can happen when people and organizations remain committed to patients with an ultra-rare disease.

To everyone who believed, persevered, advocated, researched, treated, collaborated, and refused to give up—thank you.

Today is a day to celebrate. And for the WAS community, it is the beginning of an entirely new chapter. 💙

Here is the link to the press release https://www.prnewswire.com/news-releases/ucsf-benioff-childrens-hospitals-become-the-first-us-qualified-treatment-center-contracted-to-administer-individualized-gene-therapy-waskyra-302891579.html

and the link to the Waskyra website. www.waskyra.com

UCSF Benioff Children's Hospitals, FONDAZIONE TELETHON, Therapies Accelerator, -TIGET, Aiuti, Ferrua

09/24/2026

Last chance to REGISTER. Registration closes on Friday at 5pm PDT. It's going to be an amazing, informative webinar. Click here to learn more and register: https://forms.gle/FXCXHo1pWbXegHa27

The Wiskott-Aldrich Foundation is pleased to share our September webinar. Please join our September Patient Education Webinar focused on the Prevention and Management of Infections in Wiskott-Aldrich Syndrome (WAS).

Date: Saturday, September 26, 2026
Time: 11:00 AM – 12:00 PM EDT

Webinar focus:
Infections are an important concern for individuals with WAS throughout their lives. The risk of infection and the approach to prevention and treatment can vary depending on a person's age, immune function, current health, and where they are in their treatment journey.

This webinar will provide practical guidance on the prevention and management of infections at every stage of WAS, including:
✨The role of prophylactic antibiotics, immunoglobulin replacement, and vaccinations
✨Recognizing infections that require prompt medical attention
✨Infection prevention and management before HSCT or gene therapy
✨Infection risks and management during HSCT and gene therapy
Infection prevention, immune recovery, and monitoring after HSCT or gene therapy
✨Prevention and management of infections in individuals who have not undergone HSCT or gene therapy
✨Understanding how infection risk changes over time and what precautions may continue

About Our Speaker
Dr. Alexandra Freeman
Senior Clinician
Primary Immune Deficiency Clinic
National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health (NIH)

About Our Moderator
Prof. Fabio Candotti
Head of Immunity
Lausanne University Hospital and University of Lausanne, Switzerland

Who can attend:
This webinar is designed for and open only to patients, parents, and family members with Wiskott-Aldrich Syndrome and their families (participants should be over 18 years of age).

Whether you or your child is newly diagnosed, is living with WAS without having undergone HSCT or gene therapy, is preparing for treatment, or is years beyond treatment, this session will provide important information about preventing and managing infections and staying as healthy as possible throughout the WAS journey.

We hope you will be able to join us for this meaningful conversation.

How Does Registration Work?
Registered participants will receive an email several days before the meeting containing information about joining the meeting. The email will include a link on how to join the meeting and will include phone numbers where you can join by telephone.

To register, please go to https://forms.gle/FXCXHo1pWbXegHa27
You will receive the Zoom link a few days before the session.
RSVP by Sept 23 , 2026.
If you have specific questions for the doctors, please enter them below. There will be ample time for questions and answers, and families are encouraged to submit their questions when registering.
There is no cost to attend.
We look forward to seeing you at the meeting.