SDS & Science Snapshots (2023-03-25)
In this issue: New proof-of-concept article on RNA Therapeutics and Base/Prime Editing for SDS; And, a review of diagnostic testing for...
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Blogs
Moe (age 41)
In Loving Memory Moe passed away from complications of stem cell transplantation 200 days post transplant, surrounded by his family. He...
SDS & Science Snapshots (2023-02-05)
In this issue: Unusual SDS case report from Uruguay about an adult patient; And, what exactly is a Rare Disease anyway (a project by...
SDS & Science Snapshots (2023-01-29)
In this issue: Expert consensus on the management of hematological complications in SDS by The European Society for Blood and Marrow...
SDS & Science Snapshots (2023-01-14)
In this issue: Comprehensive review article on bone marrow surveillance of SDS patients by Drs. Shimamura and Reilly; and a recap of ASH...
SDS Cell Biobank Pilot Project a success!
After the successful launch of the mouse model project last year and advancing it to phase two this year, we have expanded our efforts to...
Elijah (age 25)
In Loving Memory Elijah grew up not knowing about SDS. Neither did his family. He developed health issues around age 23 including MDS....
SDS Alliance Meets with the FDA
Dr. Eszter Hars (SDS Alliance president and CEO) joined the FDA CBER OTAT Patient-Focused Drug Development Listening Meeting.
2022 Annual Global Virtual Fundraiser - Three Million Steps Closer to #CureSDS - Huge Success Again
This October, we conducted the third annual global virtual fundraiser to support SDS research. The theme this year was THREE MILLION...
SDS Alliance meets with the White House Cancer Moonshot Team
September 30th is Rare Cancer Day. We marked the day by taking action and meeting with the White House Cancer Moonshot initiative in...
SDS Alliance is Awarded JumpStart Grant for iPSC Development
We are so honored and excited to announce that we won the JumpStart Research Tools Matching Grant through The Orphan Disease Center (ODC)...
Mouse Model Project Update: Phase I complete!
We are happy to report that Jackson Laboratory has completed the first phase of the project. They have created mice in which a large segment
SDS & Science Snapshots (2022-08-21)
In this issue: What is Congenital Exocrine Pancreatic Insufficiency; Can genetic disorders be treated in the womb? A case report in...
SDS & Science Snapshots (2022-07-31)
In this issue: What is a protein 3D structure and why it matters; New article on the structure and dynamics of the SBDS protein; DeepMind...
SDS & Science Snapshots (2022-07-23)
In this issue: What is neutropenia and how does it happen in SDS; Study highlights the multibillion dollar burden of rare disease Welcome...
SDS & Science Snapshots (2022-07-10)
In this issue: CRISPR-Cas9 turns 10; The promise of Gene Therapy for Rare Disease and Shwachman-Diamond Syndrome (SDS) Welcome to our...
SDS & Science Snapshots (2022-07-03)
In this issue: IRDiRC Working Group Recommendations for rare disease patients; The Access to Rare Indications Act could be a game changer...
SDS & Science Snapshots (2022-06-25)
In this issue: Rare-X report provides update on the true number of rare diseases; new review article on bone marrow failure disorders by...
SDS & Science Snapshots (2022-05-08)
In this issue: Dr. Bezzerri provides an update on a therapeutic approach targeting premature termination codon (PTC) mutations in SDS, or...
![Ángel Leonardo's SDS Story from Mexico [Spanish and English]](https://static.wixstatic.com/media/fb8837_e3d8245c5b1b40a6b45a31d0c2de496f~mv2.jpg/v1/fill/w_454,h_341,fp_0.50_0.50,q_90,enc_auto/fb8837_e3d8245c5b1b40a6b45a31d0c2de496f~mv2.jpg)
Ángel Leonardo's SDS Story from Mexico [Spanish and English]
"My parents taught me that apart from receiving help, I must also help. I have a big heart and I am a teacher of life for my family, a...
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