Updates

June 30, 2020June 26, 2020
The Simple Joy of Taking a Walk

We live in Illinois, where the weather can be unpredictable. Some days we have perfect weather — 70 degrees and sunny — followed immediately ... Read more
June 30, 2020June 30, 2020
AMT-130, uniQure’s Gene Therapy, Given to First Patient Group in Trial

A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington’s disease has “treated” its first two patients, uniQure announced. One person was given AMT-130 ... Read more
June 23, 2020June 19, 2020
It’s Not About You, Caregivers

I am messy. I’ve always been messy. My desk at work looks like an explosion of papers. I love to read, so it’s not ... Read more
June 23, 2020June 23, 2020
German Researcher Promotes Non-drug Alternatives for Treating Huntington’s

At least three major pharmaceutical companies are looking into potential gene therapies for Huntington’s disease — a rare inherited condition without a cure or ... Read more
June 22, 2020June 22, 2020
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization ... Read more

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May 23, 2016May 23, 2016

Blocking 2 Enzymes Seen to Reduce Nerve Cell Loss in Fruit Fly Model of Huntington’s

News

A collaboration between researchers at the University of Leicester, U.K., and the University of Maryland School of Medicine, Baltimore, may lead to a way of reversing some symptoms of neurogedenerative diseases, like Huntington’s, by blocking two specific enzymes of the kynurenine ... Read more

Updates

The Simple Joy of Taking a Walk

AMT-130, uniQure’s Gene Therapy, Given to First Patient Group in Trial

It’s Not About You, Caregivers

German Researcher Promotes Non-drug Alternatives for Treating Huntington’s

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Cooking Reminds Me of Cherished Time with My Mother

Overcoming Denial in the Face of Huntington’s Disease

Austedo to Manage Chorea Safe at Higher Doses, Study Finds

To Join or Not to Join, That Is the Question

Coalition Will Address Racial Disparities in Rare Disease Communities

Subscribe to our mailing list

The Real World Is Precious

‘Hero’ Proteins Protect Other Proteins from Stress, Research Finds

Pepinemab Clinical Trial Ongoing; Top-line Results Coming Later this Year

A Caregiver’s Biggest Lesson

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

Feeling Helpless Vs. Being Helpful

China Approves Austedo for Huntington’s-related Chorea

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Pulled Between Two Lives

Music Transports Me Back to Those Dark Diagnosis Days

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

The Inexhaustible Reality of Love

Government Agencies Approve MMJ’s Import of Marijuana for Research

Honoring a Huntington’s Family Member Dedicated to Finding a Cure

Huntington’s Disease Awareness Month Goes Virtual in May Due to Pandemic

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

Practicing Self-care and Letting Go of the Guilt

AskBio Acquires BrainVectis and Potential Gene Therapy for Huntington’s

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

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

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

Blocking 2 Enzymes Seen to Reduce Nerve Cell Loss in Fruit Fly Model of Huntington’s

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The Simple Joy of Taking a Walk

AMT-130, uniQure’s Gene Therapy, Given to First Patient Group in Trial

It’s Not About You, Caregivers

German Researcher Promotes Non-drug Alternatives for Treating Huntington’s

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Cooking Reminds Me of Cherished Time with My Mother

The Day I Summoned the Courage to Update My LinkedIn Profile

Overcoming Denial in the Face of Huntington’s Disease

Austedo to Manage Chorea Safe at Higher Doses, Study Finds

To Join or Not to Join, That Is the Question

Coalition Will Address Racial Disparities in Rare Disease Communities

Subscribe to our mailing list

The Real World Is Precious

‘Hero’ Proteins Protect Other Proteins from Stress, Research Finds

Pepinemab Clinical Trial Ongoing; Top-line Results Coming Later this Year

A Caregiver’s Biggest Lesson

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

Feeling Helpless Vs. Being Helpful

China Approves Austedo for Huntington’s-related Chorea

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Pulled Between Two Lives

Music Transports Me Back to Those Dark Diagnosis Days

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

The Inexhaustible Reality of Love

Government Agencies Approve MMJ’s Import of Marijuana for Research

Honoring a Huntington’s Family Member Dedicated to Finding a Cure

Huntington’s Disease Awareness Month Goes Virtual in May Due to Pandemic

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

Practicing Self-care and Letting Go of the Guilt

Hugging Is One of the Things I Miss the Most

AskBio Acquires BrainVectis and Potential Gene Therapy for Huntington’s

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

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

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

Blocking 2 Enzymes Seen to Reduce Nerve Cell Loss in Fruit Fly Model of Huntington’s

Pin It on Pinterest