Disability treatment – Philippine Cerebral Palsy http://philippinecerebralpalsy.org/ Tue, 24 May 2022 11:45:16 +0000 en-US hourly 1 https://wordpress.org/?v=5.9.3 https://philippinecerebralpalsy.org/wp-content/uploads/2021/11/cropped-icon-32x32.png Disability treatment – Philippine Cerebral Palsy http://philippinecerebralpalsy.org/ 32 32 Neuronascent Receives Rare Pediatric Drug Designation from FDA for NNI-351 Treatment of Fragile X Syndrome https://philippinecerebralpalsy.org/neuronascent-receives-rare-pediatric-drug-designation-from-fda-for-nni-351-treatment-of-fragile-x-syndrome/ Tue, 24 May 2022 10:00:00 +0000 https://philippinecerebralpalsy.org/neuronascent-receives-rare-pediatric-drug-designation-from-fda-for-nni-351-treatment-of-fragile-x-syndrome/

Neuronascent, Inc.

ROCKVILLE, Md., May 24, 2022 (GLOBE NEWSWIRE) — Neuronascent Inc., a clinical-stage biopharmaceutical company discovering and developing neural-generating therapies for rare developmental disorders, today announced that the Food and Drug Administration (FDA) of the United States had granted rare pediatric designation for NNI-351 for fragile X syndrome.

Fragile X syndrome (FXS) is an inherited monogenic disorder and is a major cause of intellectual disability and autism. Currently, there are no effective therapies to treat hippocampus-related deficits such as social anxiety, learning disabilities and hyperactivity as well as impaired neurogenesis.

Evaluation of NNI-351 in preclinical models of FXS (supported in part by the FRAXA organization, https://www.fraxa.org) by selectively inducing neural progenitor cells to become neurons, i.e. neurogenesis, NNI-351 reverses behavioral deficits to normal levels. Neuronascent’s oral therapy could therefore slow and potentially reverse the behavioral deficits of FXS in young people.

With a marketing application for NNI-351 for young FXS patients, Neuronascent as a sponsor will request the award of a good priority rare pediatric disease. The FDA’s Rare Pediatric Disease Priority Voucher Program is designed to incentivize companies to develop new therapies to treat rare disorders with fewer than 200,000 people and that primarily affect those under the age of 18. The priority review voucher could then be used for a subsequent drug, or can be sold to another pharmaceutical company, which speeds time to market and provides greater value for rare pediatric treatment, even with a small pool of population.

“Receiving this rare pediatric designation from the FDA allows Neuronascent to become eligible for the Pediatric Priority Review Voucher, which brings significant value to our first-in-class therapeutic NNI-351 for FXS in young people,” said the founder and CEO, Judith Kelleher-Andersson, PhD. “With a large unmet need for new therapies that can directly address pediatric behavioral deficits in FXS, for example to improve the education and social outcomes of these young patients, this designation highlights the true potential of NNI-351. “

About NNI-351

NNI-351 is Neuronascent’s patented new lead chemical entity aimed at reversing developmental disorders by producing new neurons after birth to enhance neurogenesis that is slowed during the early development of diseases such as FXS. The ability of NNI-351 to reverse behaviors in a number of models of developmental disorders, including the rare pediatric disorder, FXS, suggests this new therapy may be the first therapy to show true clinical benefit in young patients. with FXS.

About Neuronascent, Inc.

Neuronascent, Inc., a private, clinical-stage pharmaceutical company, was founded to discover and develop new therapies that treat CNS disorders with high unmet needs by replacing and enhancing the number of neurons, not just connections. Through its proprietary phenotypic discovery platform, Neuronascent has discovered a pipeline of patented small molecule neuron regeneration candidates, including clinical therapy NNI-362 for age-related disorders, particularly Alzheimer’s and of Parkinson’s.

Neuronascent website: https://www.neuronascent.com

Contact information :

Judith Kelleher-Andersson, PhD,


Such. 1-240-876-7496

Nick Veronico


Safe Harbor Statement
This release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified by words such as “would”, “may”, “will, “expects” and other terms with a similar meaning. Forward-looking statements are based on current beliefs, assumptions and expectations and speak only as of the date of this release and involve risks and uncertainties that could cause actual results to differ materially from current expectations.

Anti-TNF for the treatment of Dupuytren’s disease https://philippinecerebralpalsy.org/anti-tnf-for-the-treatment-of-dupuytrens-disease/ Mon, 23 May 2022 19:21:25 +0000 https://philippinecerebralpalsy.org/anti-tnf-for-the-treatment-of-dupuytrens-disease/

The Rheumatology Network interviewed James Woody, MD, PhD, to discuss recent positive results from treating Dupuytren’s disease with tumor necrosis factor (TNF). Woody is the CEO of 180 Life Sciences.

Dupuytren’s disease, a localized autoimmune fibrotic process, begins as a lump in the palm of the hand and moves the fingers together over time until they twitch. The wasting disease, if left untreated, can render patients unable to perform common tasks, such as tying their shoes, and may ultimately lead to surgery to correct the condition.

Current treatment plans use a “wait and see” approach until there is a finger twitch. Unfortunately, at this time, the patient is already struggling with a disability. “My wife went through that with an entire year of steroid injections, which did nothing,” Woody said.

Jagdeep Nanchahal, BSc, PhD discovered that these unusual cells, called myofibroblasts, were driven by the cytokine TNF. When he injected these nodules with anti-TNFs, Nanchahal was able to block this process and potentially prevent disease progression.

In the recent phase 2b trial, 140 patients received 4 injections of anti-TNF or placebo 3 months apart. The main criterion was the hardness of the nodule and its evolution after 1 year. The investigators found that the nodules were much softer after the anti-TNF injections. The nodule was still softer at 9 months after the last dose. The secondary endpoint was whether the nodule had increased in size. The results indicated that at 12 months his height had decreased by 39% and continued to decrease by 52% at 18 months. This is clinically significant because “some studies show that the size of the nodule over time predicts whether or not a person will have contracted fingers,” says Woody.

See the full interview below:

Long-term Covid symptoms and treatment: what we know so far https://philippinecerebralpalsy.org/long-term-covid-symptoms-and-treatment-what-we-know-so-far/ Sat, 21 May 2022 16:06:40 +0000 https://philippinecerebralpalsy.org/long-term-covid-symptoms-and-treatment-what-we-know-so-far/

Among the many confounding aspects of the coronavirus is the spectrum of possible symptoms, as well as their severity and duration. Some people develop mild illness and recover quickly, with no lasting effects. But studies estimate that 10-30% of people report persistent or new medical problems months after their first coronavirus infections – a constellation of symptoms known as long Covid. People who suffer from mild to moderate illness, as well as those who have no underlying medical conditions, can still experience debilitating long-term symptoms, including fatigue, shortness of breath, erratic heartbeat, headaches, dizziness, depression, and memory and memory problems. concentration.

These persistent medical problems are so varied that a study by a patient-led research group assessed 203 symptoms that can fluctuate or even appear out of the blue after people seem to recover.

As Dr. Ziyad Al-Aly, head of research and development at the VA St. Louis Healthcare System and clinical epidemiologist at Washington University in St. Louis, said, “If you saw a patient with a long Covid, you’ve seen a patient with long Covid.

There is little consensus on the exact definition of long Covid, also known by the medical term PASC, or post-acute sequelae of Covid-19. While the World Health Organization says the long Covid begins three months after the first episode of illness or a positive test result, the Centers for Disease Control and Prevention pegs the timeline at just over a month.