Month: September 2021

September 30, 2021September 30, 2021

Navigating a Career With Early-onset Parkinson’s Disease

Parkinson’s News Today Columns

When a person is diagnosed with Parkinson’s disease at a young age, many thoughts swirl through their head. Once the dust starts to settle, they’ll likely start thinking about their ... Read more

September 30, 2021September 30, 2021

Dragonflies, Tiny Tables, and Big Feelings: Exploring Sexual Wellness Accessibility

SMA News Today Columns

I have a fear of falling. It’s not from the great heights of suspension bridges or tall buildings. No, it’s much more humble than that. I fear I will fall ... Read more

September 30, 2021October 1, 2021

Gene Therapy SGT-001 Improves Lung Function in DMD Boys in Trial

Muscular Dystrophy News

Treatment with SGT-001 — Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) — improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD ... Read more

September 30, 2021September 30, 2021

ALS Association Offering Doctors ‘thinkALS’ Guide to Speed Diagnosis

ALS News Today, COVID-19 News

The ALS Association has launched a new diagnostic guide — called thinkALS — to aid neurologists in more quickly suspecting and diagnosing amyotrophic lateral sclerosis (ALS) at its early stages. Early diagnosis will ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Porphyria News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Hemophilia News Today

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

FAP News Today

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Cushing's Disease News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021October 1, 2021

Oct. 4 Online Conference Offers Latest Insights Into Progressive MS

Multiple Sclerosis News Today

An upcoming conference focused on progressive multiple sclerosis (MS) will offer the latest insights about the underlying biology of the disease, and treatment and clinical strategies in development. The one-day ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Angioedema News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021October 1, 2021

Enzyme Replacement Trial Ended Early After Failing to Meet Goals

Sanfilippo News

A clinical trial extension study involving 12 children with Sanfilippo syndrome type A was terminated early, after efficacy analyses found that the investigational enzyme replacement therapy it was testing failed to ... Read more

September 30, 2021September 30, 2021

Epigenetic Changes to DNA May Be Linked to PWS Symptoms

Prader-Willi News

Specific epigenetic modifications — chemical marks added to DNA that affect the way genes work — are found in people with Prader–Willi syndrome (PWS), and may affect how the disease ... Read more

September 30, 2021September 30, 2021

Network Makes Progress in California, Adds Goals for Patient Care

Sickle Cell Disease News

Two years since its establishment, the Networking California for Sickle Cell Care initiative has seen increasing numbers of specialized clinics and healthcare professionals knowledgeable about sickle cell disease (SCD) and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Batten Disease News

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

Fragile X News Today

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more