Spinraza for Most SMA Patients in China & Ben Mattlin's Book on Inter-abled Relationships

Spinraza for Most SMA Patients in China & Ben Mattlin's Book on Inter-abled Relationships

We discuss how China has approved Spinraza for most SMA patients. Also, Community Editor Kevin Schaefer reads from his review of a book about inter-abled relationships by Ben Mattlin. Mattlin is a Los Angeles-based writer with SMA Type 2, and this review is available on our main website and on our forums. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com

Episoder(443)

EMBRACE Trial Supports Spinraza’s Benefits in Broad Range of SMA Patients

EMBRACE Trial Supports Spinraza’s Benefits in Broad Range of SMA Patients

SMA News Today’s multimedia associate, Price Wooldridge, discusses the EMBRACE Trial which supports Spinraza’s benefits in a broad range of Spinal Muscular Atrophy (SMA) patients. Also, DeAnn Runge w...

15 Feb 202112min

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

SMA News Today’s multimedia associate, Price Wooldridge, discusses the National Organization for Rare Disorders (NORD) 6th “state report card”, noting progress, but raising concerns. Plus, closing in...

12 Feb 20217min

Rare Disease Groups, Pharmas Join SMA Europe Push for Newborn Screening

Rare Disease Groups, Pharmas Join SMA Europe Push for Newborn Screening

SMA News Today’s multimedia associate, Price Wooldridge, discusses rare disease groups and pharmaceutical companies, joining SMA Europe in a push for newborn screening for Spinal Muscular Atrophy (SMA...

10 Feb 20217min

Motor Function, Breathing Important in Choosing Treatment

Motor Function, Breathing Important in Choosing Treatment

SMA News Today’s multimedia associate, Price Wooldridge, discusses improvement in motor function, breathing, and ease of administration are important factors when Spinal Muscular Atrophy (SMA) patient...

8 Feb 20218min

More Severe COVID-19 Symptoms Seen in Children with SMA Type 1

More Severe COVID-19 Symptoms Seen in Children with SMA Type 1

SMA News Today’s multimedia associate, Price Wooldridge, discusses more severe COVID-19 symptoms being seen in children with Spinal Muscular Atrophy (SMA) Type 1. Also, vlogger and forums moderator D...

5 Feb 20217min

Better Ways of Capturing Progression in Types 2 and 3 Identified in Study

Better Ways of Capturing Progression in Types 2 and 3 Identified in Study

SMA News Today’s multimedia associate, Price Wooldridge, discusses better ways of capturing progression of motor abilities in Spinal Muscular Atrophy (SMA) Types 2 and 3. Forums Director Kevin Schaef...

3 Feb 202110min

#75: Alvaro Cheherlian, Founder and CEO of Wrekt Svpply

#75: Alvaro Cheherlian, Founder and CEO of Wrekt Svpply

In episode 75, host Kevin Schaefer talks with Alvaro Cheherlian from Sylmar, California. Alvaro is the founder and CEO of Wrekt Svpply, a clothing brand company. He talks about building his business, ...

2 Feb 202138min

Cure SMA Booklet Outlines Possible Risks, Benefits of Combining Treatments

Cure SMA Booklet Outlines Possible Risks, Benefits of Combining Treatments

SMA News Today’s multimedia associate, Price Wooldridge, discusses a Cure SMA booklet which outlines the possible risks and benefits of combining treatments for Spinal Muscular Atrophy (SMA). Also, ...

1 Feb 20216min

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