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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

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

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?

Targeted NGS Seen As Best When Screening For SMA And Its Carriers

Targeted NGS Seen as Best When Screening for SMA and Its Carriers

Caregivers Need Information About Help With Future Care, Study Finds

Caregivers Need Information About Help With Future Care, Study Finds

Once-Daily Risdiplam Beneficial In Patients With Types 2 And 3 SMA: Lancet

Once-Daily Risdiplam Beneficial in Patients With Types 2 and 3 SMA: Lancet

Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor Problems

Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor Problems

Developing Motor Neurons In SMA Show Altered Protein Production

Developing Motor Neurons in SMA Show Altered Protein Production

Family History Can Be Key In Identifying Silent Carriers Of SMA

Family History Can Be Key in Identifying Silent Carriers of SMA

STR1VE-EU Trial Shows Efficacy Of Onasemnogene Abeparvovec In Spinal Muscular Atrophy

STR1VE-EU Trial Shows Efficacy of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Metabolic Urine Analysis May Help Diagnose, Predict SMA

Metabolic Urine Analysis May Help Diagnose, Predict SMA

Safety And Efficacy Data Published For Novel Nusinersen Drug Delivery Method For Spinal Muscular Atrophy Patients

Safety and Efficacy Data Published for Novel Nusinersen Drug Delivery Method for Spinal Muscular Atrophy Patients

Mothers Want To Improve Children’s Quality Of Life

Mothers Want to Improve Children’s Quality of Life

FDA Lifts Partial Clinical Hold On Novartis’ SMA Drug Programme

FDA Lifts Partial Clinical Hold on Novartis’ SMA Drug Programme

New Components For Antisense Gene Therapy Show Promise In Treating Spinal Muscular Atrophy

New Components for Antisense Gene Therapy Show Promise in Treating Spinal Muscular Atrophy

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

FDA-Approved Treatment Provides Life-Saving Miracle For Girl With Spinal Muscular Atrophy

FDA-Approved Treatment Provides Life-Saving Miracle for Girl With Spinal Muscular Atrophy

Baby First At UNC To Receive Gene Therapy For SMA, Thanks To Early Check Newborn Screening

Baby First at UNC to Receive Gene Therapy for SMA, Thanks to Early Check Newborn Screening

A ‘Miracle’: Spinraza Gives Boy, Now 5, A Chance To Grow Up

A ‘Miracle’: Spinraza Gives Boy, Now 5, a Chance to Grow Up

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

Spine Surgery Effective In SMA Type 1 Children, But Not Without Risks

Spine Surgery Effective in SMA Type 1 Children, But Not Without Risks

A New Way To See Muscle Wasting In Spinal Muscular Atrophy

A New Way to See Muscle Wasting in Spinal Muscular Atrophy

Gene Therapy Improves Muscle Function For Children With Spinal Muscular Atrophy

Gene Therapy Improves Muscle Function for Children With Spinal Muscular Atrophy

DNA From Saliva Has Potential For Detecting SMA

DNA From Saliva Has Potential for Detecting SMA

Infants With Symptomatic SMA Reach Milestones With Gene Therapy

Infants With Symptomatic SMA Reach Milestones With Gene Therapy

0 resources available: