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Spinal Muscular Atrophy

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Spinal Muscular Atrophy (SMA): A Parent's Journey from First Signs to Diagnosis

Apr 8, 2026

#370 Genetics Wrapped: 2025 Top Advances in Genomic Medicine

Dec 5, 2025

AAV. Resources on cureSYNGAP1.org. Congrats to Ultragenyx & Novartis. Conf is in 5 days! #S10e190

Nov 27, 2025

#70 The First Spinraza Patients: A Rare Mama’s Advocacy for her Son with SMA

Oct 20, 2025

270 - RDD 2025 with Jett Foundation: "Disability Doesn’t Define Us!"

Apr 7, 2025

Cell and Gene Therapy Access Challenges

Dec 4, 2024

The Coast 2 Coast Challenge benefiting the SYNGAP1 #ProMMiS for Clinical Excellence is critical. #S10e152

Oct 22, 2024

250 - Thriving with Duchenne - Rare Disease Day with Jett Foundation

Apr 22, 2024

Hemophilia Gene Therapy Redux – The End of the Beginning?

Jan 25, 2024

220 - Season Finale - How Much is Too Much?

May 29, 2023

Best Practices in the Care of Adult Patients With Spinal Muscular Atrophy

Jan 23, 2023

SYNGAP-A-PALOOZA ‘22, NASHVILLE & a bit more on ASOs #S10e81

Nov 10, 2022

#207 N-Lorem: Dr. Sessions Cole on the Diagnostic Odyssey

Oct 21, 2022

A Review of Rare Conditions Across the Lifespan: Pediatric Neuromuscular Disorders

Oct 12, 2022

Rare Disease Siblings, The Glass House Children - Bulletproof and Shattered - With SMA Sibling Cara Freedman

Aug 18, 2022

Spinal Muscular Atrophy (SMA) and its Management

Jun 21, 2022

Spinal Muscular Atrophy and its Management

Jun 21, 2022

#186 n-Lorem: Nano-Rare Diseases with Luke Rosen and Wendy Chung

May 27, 2022

The Critical Role of Newborn Screening - Rare Mom Alison Breitbarth and Infantile Pompe Disease

May 12, 2022

The life-changing power of assistive technologies | Jane Velkovski

Feb 8, 2022

What Happens Now - Baby Boy is a Medical Miracle After Being Treated for Spinal Muscular Atrophy Type 1 with Kathryn Alexander

Feb 3, 2022

One of the Youngest Children to be Given Treatment for Spinal Muscular Atrophy I with Kathryn Alexander

Jan 27, 2022

Stoke Therapeutics & Acadia Pharmaceuticals are working on SYNGAP1 #S10e43

Jan 14, 2022

The Importance of Newborn Screening in Every State with ALD Alliance Founder Elisa Seeger

Dec 2, 2021

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