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- Muscular Atrophy

Muscular Atrophy Newsletter

'People didn’t believe me when I said my son was going to die' 15 Jun 2025 15:54 GMT
… " baby's spinal muscular atrophy diagnosis. She said: "It … ; He was diagnosed with spinal muscular atrophy (SMA) at four weeks old …
Redefining Fabry Disease Care: Gene Therapy and Long-Acting ERT Prepare to Disrupt Status Quo | Competitive Intellignece 15 Jun 2025 12:06 GMT
… Related Ci Reports 1. Spinal Muscular Atrophy Genetic Understanding Disease Modifying Therapies …
[Ad Hoc Announcement Pursuant To Art. 53 LR] Roche Provides Safety Update On Elevidystm Gene Therapy For Duchenne Muscular Dystrophy In Non-Ambulatory Patients 15 Jun 2025 08:49 GMT
… disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer …
Fatherhood redefined: Strength, sensitivity, and science 14 Jun 2025 23:29 GMT
… disorders such as thalassemia, spinal muscular atrophy (SMA), or cystic fibrosis helps …
Families to face 'bleak future' if children's hospices not given more funding 14 Jun 2025 21:46 GMT
… muscle-wasting condition called Spinal Muscular Atrophy (SMA) Type 2. The condition …
Jefferies Names Akero Therapeutics, Scholar Rock As Biotech Companies With Takeover Potential, But Retail Isn’t Pleased 14 Jun 2025 06:11 GMT
… investigational treatment Apitegromab in spinal muscular atrophy by late September. The company …
Singapore’s Nurulasyiqah Mohd Taha wins bronze at World Boccia Cup 13 Jun 2025 21:27 GMT
… -year-old, who has spinal muscular atrophy, clinched the bronze medal in …
Scholar Rock Reports New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) 13 Jun 2025 20:41 GMT
… apitegromab for patients with spinal muscular atrophy (SMA) and other severe and …
Cristiano Ronaldo once threw away his captain’s armband in anger; here’s how it became a life-saving moment for 6-month-old boy with rare disease 13 Jun 2025 11:23 GMT
… , who was suffering from spinal muscular atrophy. The three-day auction received …
Emirati man with rare blood disorder receives $2 million gene therapy 13 Jun 2025 04:01 GMT
… rare genetic disorders like spinal muscular atrophy and Duchenne muscular dystrophy, two … administer gene therapy for spinal muscular atrophy, and has since delivered 100 …

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