The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Articles by Patricia Inacio, PhD
Preclinical and clinical data on SRK-015 continue to support the therapy’s potential to treat spinal muscular atrophy (SMA),…
Asuragen has launched the AmplideX PCR/CE SMN1/2 Plus Kit, which according to the company provides the most comprehensive…
BillionToOne’s non-invasive prenatal blood test, which helps diagnose hereditary and rare diseases in fetuses, is now available in Germany,…
The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help…
Adding reldesemtiv to survival of motor neuron (SMN) restoration therapies — namely Spinraza (nusinersen) or the investigational…
Optimal care of children with spinal muscular atrophy (SMA) requires improving competence and an adequate exchange of knowledge among…
The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat…
A non-invasive electrophysiology technique called motor unit number index, or MUNIX, can effectively assess the loss of motor nerve cells…
Researchers at Mizzou — the University of Missouri — have started a new company, Shift Pharmaceuticals, to test…