Differences in SMA newborn screening practices in the U.S. could impact referral patterns or the timing of therapeutic ...

Children with spinal muscular atrophy (SMA) experience benefits from receiving a disease-modifying gene therapy, but many ...

Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment ...

The high cost and limited availability of treatment are still major challenges for patients with a rare genetic disorder ...

India has 8.4 to 19 crore rare disease patients, according to health ministry estimates. But current beneficiaries in most ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal ...

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

PTC-607 is under clinical development by PTC Therapeutics and currently in Phase I for Huntington Disease. According to GlobalData, Phase I drugs for Huntington Disease does not have sufficient ...

While Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market for spinal muscular atrophy (SMA) treatments, Novartis’ research chief Jay Bradner has a ...

Clinical data on 8 patients were retrospectively collected; all patients were taking either nusinersen, risdiplam, or had gene therapy. Evidence of heighted hepatic echogenicity—indicating mild ...

Suggested remit: To appraise the clinical and cost effectiveness of nusinersen and risdiplam within their marketing authorisations for treating spinal muscular atrophy.

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