Just 24 years old, Álvaro Tapia, a blind pianist from Malaga, is winning the hearts of audiences with extraordinary musical ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

Facial abnormalities (saddle nose, protuberant mandible), central nervous system involvement (mental retardation, optic nerve atrophy, seizure disorders), and bone or joint involvement (frontal ...

Data from the phase 2 HELIOS trial of AMX0035 in Wolfram syndrome – a genetic disorder that leads to diabetes, optic nerve atrophy, hearing loss, and neurodegeneration with no FDA-approved ...

Imagine spending an entire year floating in space, orbiting the Earth at 17,500 miles per hour, completely weightless. Sounds ...

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And yet, that isn’t always the case. Many eye diseases, such as geographic atrophy (GA), can progress so stealthily — and with so few symptoms — that it can take some time before you’re ...

It’s necessary for proper red blood cell production, DNA formation, and nerve function. Vitamin B12 also plays a key role in reducing levels of an amino acid called homocysteine, high levels of ...

FIREFISH (NCT02913482) in Type 1 SMA (inclusion criteria [IC]: aged 1–7 months at enrollment) SUNFISH (NCT02908685) in Types 2/3 SMA (IC: aged 2–25 years at enrollment) JEWELFISH (NCT03032172) in SMA ...