Updates

June 4, 2020June 4, 2020
Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). Included ... Read more
June 2, 2020June 2, 2020
$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to $1 million in funding for gene therapy research ... Read more
June 1, 2020May 28, 2020
What I’ve Learned from Starting a Twitch Channel

When I started my Twitch channel in February, I wasn’t sure what to expect. I became quite familiar with the video game streaming platform ... Read more
May 29, 2020June 1, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays ... Read more
May 28, 2020May 28, 2020
Let’s Aim to Achieve True Global Accessibility

On May 21, the world celebrated its ninth Global Accessibility Awareness Day, but accessibility awareness hasn’t yet risen to its deserved level of interest. ... Read more

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May 21, 2018June 3, 2019

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide ... Read more

Updates

Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

What I’ve Learned from Starting a Twitch Channel

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Let’s Aim to Achieve True Global Accessibility

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

Why Self-monitoring Our Functioning Levels Is Important

IGNITE DMD Trial Remains on Hold, FDA Requests More Gene Therapy Data

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Living Joyfully Helped Me Overcome Fear, Anger, and Sadness

Experts Offer Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic

The Power of Empathy Among Friends

CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy Work

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

PTC and Sarepta Say MD Treatment Supplies Uninterrupted, Encourage Use of Patient Support Programs

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

The Impossibility of Putting a Value on a Life

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

MDA’s Medical Advisory Team to Help Guide Research and Patient Care

COVID-19 Delays Phase 3 Trial of Givinostat in DMD, Italfarmaco Says in Webinar

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

The ‘Crip Camp’ Utopia Made Us a Better Society Today

Duchenne MD Patient Tanner Rico Approaches Life With Optimism

How to Best Live with One Another During Quarantine

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

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Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK

What I’ve Learned from Starting a Twitch Channel

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Let’s Aim to Achieve True Global Accessibility

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Subscribe to our mailing list

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

Why Self-monitoring Our Functioning Levels Is Important

IGNITE DMD Trial Remains on Hold, FDA Requests More Gene Therapy Data

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

Living Joyfully Helped Me Overcome Fear, Anger, and Sadness

Experts Offer Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic

The Power of Empathy Among Friends

CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy Work

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

PTC and Sarepta Say MD Treatment Supplies Uninterrupted, Encourage Use of Patient Support Programs

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

The Impossibility of Putting a Value on a Life

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

MDA’s Medical Advisory Team to Help Guide Research and Patient Care

COVID-19 Delays Phase 3 Trial of Givinostat in DMD, Italfarmaco Says in Webinar

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

The ‘Crip Camp’ Utopia Made Us a Better Society Today

Duchenne MD Patient Tanner Rico Approaches Life With Optimism

How to Best Live with One Another During Quarantine

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

Pin It on Pinterest