Posts by: Patricia Inacio PhD

Patricia Inacio PhD

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

April 16, 2021April 16, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

November 23, 2020November 23, 2020

Australia Offers Subsidized Coverage of Spinraza for Pre-symptomatic Children With SMA

News

Australia is expanding access to Spinraza (nusinersen) to children and infants with severe spinal muscular atrophy (SMA) who have not yet developed symptoms, a period when the medicine is likely to benefit ... Read more

July 29, 2020July 30, 2020

Plans Advance for Biomarkers Panel to Assess Drug-induced Skeletal Muscle Injury

News

A new panel of four safety biomarkers to assess acute drug-induced skeletal muscle injury in Phase 1 clinical trials has received a positive response from the U.S. Food and Drug ... Read more

July 10, 2020July 10, 2020

Asuragen’s Diagnostic Test for SMA Approved for Use in Europe

News

Asuragen’s diagnostic test for the analysis of genes associated with spinal muscular atrophy (SMA) — called AmplideX SMA Plus Kit — was given the European CE mark, approving it for commercial ... Read more

March 9, 2020May 15, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking ... Read more

February 21, 2020February 21, 2020

SRK-015 Data Continue to Support Potential for SMA, Scholar Rock Says

News

Preclinical and clinical data on SRK-015 continue to support the therapy’s potential to treat spinal muscular atrophy (SMA), developer Scholar Rock said in a press release. Recruitment for a Phase ... Read more

November 15, 2019November 17, 2019

New Screening Kit Quickly and Thoroughly Analyzes SMN1, SMN2 Genes in Lab, Asuragen Says

News

Asuragen has launched the AmplideX PCR/CE SMN1/2 Plus Kit, which according to the company provides the most comprehensive analysis of genes associated with spinal muscular atrophy (SMA) on the market ... Read more

November 8, 2019November 17, 2019

Prenatal Blood Test for SMA Now Available in Four Countries in Europe, BillionToOne Announces

News

BillionToOne’s non-invasive prenatal blood test, which helps diagnose hereditary and rare diseases in fetuses, is now available in Germany, Austria, Switzerland, and the Netherlands, the company announced. The test, which ... Read more

July 15, 2019August 20, 2019

Prenatal Blood Test for SMA and Other Single-Gene Diseases Now Available, BillionToOne Announces

News

The diagnostics company BillionToOne has launched its first product for commercial and clinical use — a blood test designed to help diagnose hereditary and rare diseases in fetuses. Called UNITY, the test ... Read more

July 10, 2019November 19, 2019

Adding Reldesemtiv to Spinraza or Investigational SMN-C1 Improved Muscle Function in SMA Mouse Model

News

Adding reldesemtiv to survival of motor neuron (SMN) restoration therapies — namely Spinraza (nusinersen) or the investigational SMN-C1 (an analogue to risdiplam) — can improve muscle function in a mouse ... Read more

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