Norman Putzki, MD, Global Development Head of Neuroscience and Gene Therapy at Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for spinal muscular atrophy (SMA).

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#CheckRare #SMA #RareNeurology #RareMusculoskeletal

Sonia Gobeil, co-founder of Ataxia of Charlevoix-Saguenay Foundation, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS).

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#CheckRare #RareNeurology #RareMusculoskeletal #ARSACS

FDA Approves Myasthenia Gravis Treatment Nipocalimab The U.S. Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG).

The U.S. FDA has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis.

checkrare.com/fda-approves...

#CheckRare #FDAApproval #MyastheniaGravis #RareNeurology #RareMusculoskeletal

Paul Bolno, MD, discusses positive results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with Duchenne muscular dystrophy amenable to exon 53 skipping.

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#CheckRare #DMD #RareMusculoskeletal

Aravindhan Veerapandiyan, MD, discusses results from the phase 1/2 EXPLORE44 clinical trial for Duchenne muscular dystrophy (DMD).

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#CheckRare #RareMusculoskeletal #RareNeurology #RareGenetic #DMD #Duchenne #RareDisease

Cancers There are over 200 different types of rare cancers, but only a small number of people are diagnosed with each type.

💡Rare Disease Spotlight: ALS

Learn more about this rare disease with our latest article checkrare.com/diseases/can...

#CheckRare #RareDisease #ALS #RareMusculoskeletal #RareNeurology

Results From the Phase 3 Vivacity-MG3 Study in Myasthenia Gravis Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with MG.

Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with generalized myasthenia gravis (MG).

checkrare.com/results-from...

#CheckRare #MyastheniaGravis #RareNeurology #RareMusculoskeletal

The U.S. Food and Drug Administration has approved vimseltinib for adult patients with symptomatic tenosynovial giant cell tumor (TGCT).

checkrare.com/fda-approves...

#FDAApproval #TGCT #RareMusculoskeletal