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SMA Press
About SMA: SMA Press
The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one…
Read MoreToday Scholar Rock shared a community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA: Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular…
Read MoreNEW YORK — March 7, 2005 – Two-year-old Owen Cain cannot lift his head or move his limbs. He’s fed through a tube, and even the simple act of sitting in a chair can trigger an alarming breathing spasm. The child has Spinal Muscular Atrophy, or SMA – the leading genetic killer of infants and…
Read MoreBy Gina Shaw They don’t have MD or PhD degrees. They don’t run research laboratories, direct hospitals, or oversee major academic medical centers. Yet they have helped changed the course of neurological research, driving tens of millions of research dollars – both private and federal – into the quest for treatments and cures for diseases…
Read MoreWhen Ellen Goldstein of Brooklyn gave birth last November to her only child, Owen, medical tests offered no clues that five months later he would be crippled by a deadly and irreversible genetic disease. Tests shortly before Owen was born revealed no abnormalities, and a physical evaluation right after his birth showed he was in…
Read MoreFifty years ago today, James Watson’s and Francis Crick’s scientific paper ignited a revolution in modern science. Fidgeting with cardboard cutouts and X-ray photographs, Watson and Crick discovered the spiral staircase structure of DNA. Their description of the double-helix sparked a torrent of research into DNA that today has us on the verge of finding…
Read MoreBy Loren A. Eng Fifty years ago, Francis Crick and James Watson ignited a revolution in modern science. Toying with a collection of cardboard cutouts and X-ray photographs, the two scientists discovered the structure of deoxyribonucleic acid, known as DNA. Their spontaneous observation of the “double helix” sparked a torrent of research into DNA that…
Read MoreABC NEWS UPCLOSE: ARYA SINGH ANCHORED BY: TED KOPPEL WITH GUESTS: LOREN ENG AND DINAKAR SINGH JANUARY 21, 2003 TED KOPPEL, ABC NEWS (OC) “Up Close” tonight, a young girl, a two-year-old, whose name is Arya. She lives in New York City, with her parents and baby brother. Arya has a genetic disease that the…
Read MoreBy ADAM COHEN DECEMBER 29, 2002 When Loren Eng, a briskly efficient Stanford Business School graduate, lobbies Congress for research funds for the disease that is crippling her 2-year-old daughter, she takes along almost everything she needs to make an airtight case. She has figures showing that the disease, spinal muscular atrophy, is badly underfinanced…
Read MoreNew York, WABC, September 19, 2002 — For parents, few things are more difficult than watching their child struggle with a serious illness. When a muscle disease threatened their daughter, a frustrated family looked for a treatment and then found a way to help others facing the same crisis. Now, ABC7’s On Call with Dr.…
Read MoreColumbia University Press: “Columbia University & Columbia-Presbyterian Med Ctr Open New SMA Clinic”
WHEN & WHERE: Tuesday, September 17, Noon Harkness Pavilion 180 Fort Washington Avenue New York NY WHAT: The Departments of Neurology Columbia University and at Columbia Presbyterian Medical Center are celebrating the opening of the SMA (spinal muscular atrophy) Clinic, a center devoted to the treatment and management of a group of diseases that affect…
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