Health ❯ Genetics

Rare Diseases

Progeria Cerebellar Ataxia Sanfilippo Syndrome CRISPR Technology Activated PI3-Kinase Delta Syndrome Congenital Disorders Neuroscience Muscle Disorders PPFIBP1 Mutation Situs Inversus Leber Congenital Amaurosis Genome Sequencing Diagnostic Testing Genetic Disorders Movement Disorders Therapeutics Prader-Willi Syndrome

Soleno Therapeutics Sued in Federal Securities Class Action Over DCCR Safety Disclosures

Investors have until May 5, 2026 to seek lead-plaintiff status in a case alleging misstatements about the Prader–Willi syndrome drug’s risks.

Proteomic Test Cuts Childhood Rare Disease Diagnosis to Under Three Days

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