The Cure SMA educational campaign is designed to equip parents and healthcare professionals with the tools needed to recognize motor delays in spinal muscular atrophy (SMA) patients early, allowing infants ... Read more
AveXis‘ gene therapy AVXS-101, now under review for approval to treat infants with spinal muscular atrophy (SMA) type 1 in the U.S. and Europe, may be truly “transformative,” said Kenneth Hobby, president of ... Read more
Cytokinetics and Cure SMA have expanded their existing partnership to increase education, awareness and fundraising for spinal muscular dystrophy (SMA). As a National Platinum Partner for 2018, Cytokinetics will ... Read more
Cure SMA will host the world’s largest regular gathering on spinal muscular atrophy next month in Dallas, bringing together leading SMA researchers and clinicians, as well as families living with the ... Read more
If 8-year-old Branden Lim had been born in Australia instead of Malaysia, his parents soon would have no problem obtaining Spinraza (nusinersen) to treat his spinal muscular atrophy (SMA). Australian ... Read more
SMA expert Dr. Arthur Burghes is calling for newborn screening for spinal muscular atrophy, and the urgent approval of new therapies for treating the disease.
Burghes’ remarks came in a ... Read more
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