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SMA Press


About SMA: SMA Press

Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with Type 1 SMA

January 23, 2020

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced positive data from the pivotal Part 2 of FIREFISH, a study evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The study met its primary endpoint of a statistically significant proportion of infants sitting without support for at least five seconds after one year…

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Scholar Rock announces completion of TOPAZ trial enrollment

January 10, 2020

Scholar Rock announced today that enrollment of its TOPAZ Phase 2 trial of SRK-015 in patients with Type 2 and Type 3 SMA is complete. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. For more information, please see the press release from Scholar…

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Neurology Today: “Advocacy for Neurological Disease: The ‘Family Business’ No Family Wants”

January 5, 2004

By 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…

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The New York Times: “A Deadly Disease of Infants Attracts New Research Money,” by Anahad O’Connor

October 28, 2003

When 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…

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North County Times: “Cure for Genetic Disease Requires Money,” by George Eng

April 25, 2003

Fifty 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…

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Pittsburgh Post Gazette: “Funding Needed to Cure a Disease that Makes Children Waste Away”

March 18, 2003

By 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…

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Parents Magazine: Parents Support Circle, “Arya, 3, Has Spinal Muscular Atrophy”

March 4, 2003
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Nightline: UpClose with Ted Koppel. “Arya Singh.”

January 21, 2003

ABC 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…

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The New York Times: “When Not Knowing a Celebrity Could Prove Fatal”, by Adam Cohen

December 29, 2002

By 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…

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WABC-TV, New York: “Family Helping Others Learn More About SMA.”

September 19, 2002

New 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.…

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Columbia University Press: “Columbia University & Columbia-Presbyterian Med Ctr Open New SMA Clinic”

September 17, 2002

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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