Scholar Rock: More Than Just An SMA Biotech With Q2 2025 Obesity Treatment Data
Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for Priority Review to be given. Explore more details here.
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Which Is a Better Investment, Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. Stock?
Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.
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Biogen’s higher dose spinal muscular atrophy regimen accepted for review by EMA/FDA
Biogen has announced that the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have started ...
MDA Launches 75th Anniversary Campaign and Premieres New PSA to Shine a Light on Rare Diseases
Celebrating 75 Years of Progress, Legacy, Impact, and Momentum Leading up to Rare Disease Day on February 28 Muscular ...
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Spinal cord gray matter may be imaging marker of SMA types 2, 3
More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...
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Battle against the body's own nerves: How Motor Neurone Disease alters lives
MNDs are neurological disorders that destroy motor neurons, the cells that control skeletal muscle activity such as walking, breathing, speaking, and swallowing.
People with spinal muscular atrophy demand urgent action from the Centre to reduce medicine costs
Health activists demand government action to reduce the cost of SMA medicines, citing constitutional obligations and ...
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Biogen Seeks FDA Approval for a Higher Dosing Regimen of Spinraza for Spinal Muscular Atrophy
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
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Spinal muscular atrophy: Patients, activists seek health ministry’s intervention to bring down medicine cost
The health activists argue that the government is empowered with effective law and policy tools and therefore should ...
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Centre abdicates its constitutional obligation, criticises patients and experts on SMA issue
Centre abdicates its constitutional obligation, criticises patients and experts on SMA issue: Our Bureau, New Delhi Friday, January 24, 2025, 18:15 Hrs [IST] Patients and public h ...
FDA & EMA Accept Biogen's Filings for Higher Dose SMA Drug
BIIB's regulatory filings are supported by late-stage data, which show that treatment with a higher Spinraza dose improves motor function in infants with SMA.