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Trusted Resources: News & Meetings

Latest announcements and gatherings

18 resources available:

Dr John Brandsema Highlights Progress In SMA Treatment And Improved Outcomes For Patients

Dr John Brandsema Highlights Progress in SMA Treatment and Improved Outcomes for Patients

Biohaven Enrolls First Patient In Phase 3 Trial Of Taldefgrobep Alfa In Spinal Muscular Atrophy (SMA)

Biohaven Enrolls First Patient in Phase 3 Trial of Taldefgrobep alfa in Spinal Muscular Atrophy (SMA)

Roche’s Evrysdi Offers Survival Benefits In Babies With SMA

Roche’s Evrysdi Offers Survival Benefits in Babies With SMA

Biogen Announces New Updates Across Its SMA Research Program At 2022 MDA Conference

Biogen Announces New Updates Across Its SMA Research Program at 2022 MDA Conference

Will Parental Vaccine Hesitancy Retard The Embrace Of Life-Saving Newborn Genetic Screening And Emerging Gene Therapy Revolution?

Will Parental Vaccine Hesitancy Retard the Embrace of Life-Saving Newborn Genetic Screening and Emerging Gene Therapy Revolution?

FDA Grants Evrysdi® Priority Review Based On Results From Treating Pre-Symptomatic Infants With Spinal Muscular Atrophy

FDA Grants Evrysdi® Priority Review Based on Results From Treating Pre-Symptomatic Infants With Spinal Muscular Atrophy

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6 resources available:

Scholar Rock Provides Corporate Update And Highlights Priorities For 2022

Scholar Rock Provides Corporate Update and Highlights Priorities for 2022

Biogen Exercises Option With Ionis To Develop And Commercialize Investigational ASO For SMA

Biogen Exercises Option With Ionis to Develop and Commercialize Investigational ASO for SMA

Why Some Drugs Cost $2.1 Million Per Dose And How One Company Plans To Change This

Why Some Drugs Cost $2.1 Million per Dose and how one Company Plans to Change This

A Dallas Startup Aims To Build A Big Business—and Transform Lives—by Curing Rare Diseases

A Dallas Startup Aims to Build a Big Business—and Transform Lives—by Curing Rare Diseases

Biogen To Launch Study Of Higher Spinraza Dose In Patients With Later Onset SMA

Biogen to Launch Study of Higher Spinraza Dose in Patients With Later Onset SMA

Cure SMA, Cytokinetics Renew Joint Efforts To Raise Funds And Awareness

Cure SMA, Cytokinetics Renew Joint Efforts to Raise Funds and Awareness

9 resources available:

Girl Born With Progressive Disease SMA On The Mend, Has Big Plans For The Future

Girl Born With Progressive Disease SMA on the Mend, Has Big Plans for the Future

A New Hip For A Patient With Rare Neuromuscular Condition

A New Hip for a Patient With Rare Neuromuscular Condition

New Treatment Can Help Fresno Infant With Rare Genetic Disorder

New Treatment Can Help Fresno Infant With Rare Genetic Disorder

Springfield Second-Grader Attends Class From Home Through Double Robotics

Springfield Second-Grader Attends Class From Home Through Double Robotics

Artist With SMA Pens Song ‘SPACES’ About Looking Beyond Disability

Artist With SMA Pens Song ‘SPACES’ About Looking Beyond Disability

How One Mother Is Raising Awareness For Spinal Muscular Atrophy – A Leading Genetic Cause Of Infant Death

How One Mother Is Raising Awareness for Spinal Muscular Atrophy – A Leading Genetic Cause of Infant Death

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11 resources available:

The Orphan Drug Act Turns 40: NORD Celebrates Its Impact on Rare Diseases

SMA Screening Now Available To 87% Of Newborns In US

SMA Screening Now Available to 87% of Newborns in US

Study Highlights Gaps In Knowledge Among Prenatal Genetic Counselors

Study Highlights Gaps in Knowledge Among Prenatal Genetic Counselors

Vitamin K Deficiency Caused Blood Clotting Problem In SMA Teen

Vitamin K Deficiency Caused Blood Clotting Problem in SMA Teen

The Real Reason Some Newborns Don’t Get Screening For Deadly Diseases

The Real Reason Some Newborns Don’t Get Screening for Deadly Diseases

Newborn Screening, Treatment Make Big Difference With Rare Muscle Disorder

Newborn Screening, Treatment Make Big Difference With Rare Muscle Disorder

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7 resources available:

2023 Annual SMA Conference

2023 Annual SMA Conference

Rare Disease Day At NIH 2023

Rare Disease Day at NIH 2023

Rare Disease Week On Capitol Hill

Rare Disease Week on Capitol Hill

Fighting For Kaiden Facebook Live: How Do I Promote An Active Lifestyle?

Fighting for Kaiden Facebook Live: How Do I Promote an Active Lifestyle?

6th Annual Faces Of SMA Celebration & Silent Auction

6th Annual Faces of SMA Celebration & Silent Auction

Paul Reed Smith Guitar Model SE 24-08

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