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Norman Putzki, MD, Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for spinal muscular atrophy. checkrare.com/positive-safet… #CheckRare #SMA #RareNeurology #RareMusculoskeletal

Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. Learn more on our Learning Page at checkrare.com/neuroblastoma/ #CheckRare #Neuroblastoma #RareCancer

The U.S. FDA approved Welireg (belzutifan) for patients ages 12 years and older with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma. checkrare.com/fda-approves-t… #CheckRare #FDAApproval #RareGenetic

💡Rare Disease Spotlight: IgG4-RD Learn more about this rare disease with our latest article checkrare.com/newly-approved… #CheckRare #RareDisease #IgG4RD #RareAutoimmune #RareSkin

This CME program, hosted by John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients who have hematologic malignancies. checkrare.com/learning/p-hem… #CheckRare #CME

Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, provides an overview of urea cycle disorders (UCDs) and discusses developing novel therapies. checkrare.com/urea-cycle-dis… #CheckRare #RareDisease #UCD #RareMetabolic

Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, discusses how industry partners can support the mental health of rare disease communities. checkrare.com/mental-health-… #CheckRare #RareDisease #MentalHealth

Johanna Rossell, Senior Vice President and General Manager of Rare Diseases at Sumitomo Pharma America, discusses best practices for navigating challenges of the orphan drug market. checkrare.com/navigating-the… #CheckRare #RareDisease #OrphanDrug

CME: Fabry Disease Research Highlights Learn more at checkrare.com/learning/p-fab… #CheckRare #CME #FabryDisease

Connie Lee, PsyD, Chief Executive Officer of Alliance to Cure Cavernous Malformations, discusses the diversity of patient advocacy group initiatives. checkrare.com/the-diversity-… #CheckRare #PatientAdvocacy #RareDiseases

Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, discusses the company’s current drug development programs. checkrare.com/camp4-therapeu… #CheckRare #RareNeurology #RareMetabolic #UCD #SYNGAP1

Larisa Geskin, MD, Columbia University Medical Center, discusses the challenges of diagnosing cutaneous T-cell lymphoma. #CheckRare #RareDisease #CTCL checkrare.com/ctcl-the-role-…

David Curren, patient advocate and board member for Breath of Hope Rhode Island, discusses his grandson’s diagnostic journey with idiopathic pulmonary hemosiderosis. checkrare.com/a-patients-dia… #CheckRare #RareLung #IPH

Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath. Learn more at checkrare.com/tenosynovial-g… #CheckRare #RareDisease #TGCT

The I CAN Study is now enrolling adults diagnosed with immunoglobulin A nephropathy (IgAN) to evaluate an investigational study treatment option that may improve kidney function and slow the progression of the disease in adults. Click here to learn more. …inicalstudy.alexionclinicaltrials.com/?utm_source=Ch…

Yuliya Linhares, MD, chief of Lymphoma Services at Miami Cancer Institute, provides an overview of cutaneous T-cell lymphoma #CTCL and discusses some strategies for shortening the diagnostic journey of this rare cancer. #CheckRare #RareDisease checkrare.com/ctcl-shortenin…

The U.S. Food and Drug Administration has recently approved the following treatments. Stay up to date with new rare disease treatments approvals at checkrare.com/2025-orphan-dr… #CheckRare #FDAApproval #RareDisease

Sanjay Ahuja, MD, Pediatric Hematologist, Innovative Hematology and the IHTC, discusses the U.S. FDA approval of Qfitlia (fitusiran) for patients with hemophilia A or B. checkrare.com/phase-3-studie… #CheckRare #RareHematology #Hemophilia

Kimberly LeBlanc, Genetic Counselor, Director of the UDN Coordinating Center at Harvard Medical School, discusses approaching variants of uncertain significance in rare diseases. checkrare.com/the-undiagnose… #CheckRare #UndiagnosedDiseaseNetwork #UDN #RareDisease

Annette Bakker, PhD, Chief Executive Officer of the Children’s Tumor Foundation, discusses repurposing shelved assets for rare diseases. checkrare.com/repurposing-sh… #CheckRare #RareDisease