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Oregon toddler gets first-of-its-kind treatment for rare disease

Lily Ahrens was diagnosed with spinal muscular atrophy, also known as SMA. It's estimated there are more than 84,000 carriers for the genetic disease. ....

United States , Karen Chen , Daniel Ahrens , Lily Ahrens , Alysa Ahrens , Muscular Atrophy Foundation , Spinal Muscular Atrophy ,

Contrasting Amphastar Pharmaceuticals (NASDAQ:AMPH) & PTC Therapeutics (NASDAQ:PTCT)

Amphastar Pharmaceuticals (NASDAQ:AMPH – Get Free Report) and PTC Therapeutics (NASDAQ:PTCT – Get Free Report) are both mid-cap medical companies, but which is the superior stock? We will compare the two businesses based on the strength of their institutional ownership, analyst recommendations, profitability, risk, earnings, dividends and valuation. Volatility and Risk Amphastar Pharmaceuticals has a […] ....

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Gene Therapy Market is Set to Soar at a Revenue of US$ 19.2 Billion By 2030 | Astute Analytica |

Gene Therapy Market is Set to Soar at a Revenue of US$ 19.2 Billion By 2030 | Astute Analytica |
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United States , Sarepta Therapeutics Inc , Drug Administration , Amgen Inc , Gilead Sciences Inc , Muscular Atrophy Foundation , National Institutes Of Health , Neurophth Therapeutics Inc , Global Gene Therapy Market , Sample Report , Gene Therapy , Rising Cancer Cases , Viral Vector Therapies , Gene Therapy Products , Generation Delivery Technologies , Global Gene Therapy , Indication Segment , Vector Segment , Gene Type Segment , Full Report , Please Follow This , America Emerges , Significant Revenue Contributor , North America , Spinal Muscular Atrophy Foundation , National Institutes ,

PTC to sell spinal muscular atrophy drug royalties for $1B

BioPharma ....

Kristen Kluska , Muscular Atrophy Foundation , Cantor Fitzgerald , Drug Administration , Blackstone Life Sciences , Dive Brief , Royalty Pharma , Spinal Muscular Atrophy Foundation , Ferring Pharmaceutical Adstiladrin ,

Royalty Pharma (RPRX) to Purchase Up to $1.5B of PTC Therapeutics' Royalty on Evrysdi

Royalty Pharma (RPRX) to Purchase Up to $1.5B of PTC Therapeutics' Royalty on Evrysdi
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United States , Wilmer Cutler Pickering Hale , Roche Evrysdi , Johnson Tremfya , Novarti Promacta , Matthew Klein , Johnson Imbruvica , Pablo Legorreta , Drug Administration , Therapeutics Inc , Goodwin Procter , Muscular Atrophy Foundation , Exchange Commission , Royalty Pharma , Chief Executive Officer , Spinal Muscular Atrophy Foundation , Adjusted Cash Receipts , Pharma Investor Relations ,